Gene therapy involves introducing genetic material into cells to treat disease. It is used for inherited disorders, cancers, and infectious diseases. Viruses are commonly used as vectors to deliver therapeutic genes, though safety issues exist. Some early successes included treating severe combined immunodeficiency using retroviruses to deliver the ADA gene ex vivo. However, the first death in gene therapy occurred treating ornithine transcarbamylase deficiency using an adenovirus vector, highlighting the risks. Improved delivery methods like liposomes may help overcome current limitations and advance this promising approach.
This document discusses gene therapy, including its introduction, types, strategies, approaches, methods, target cells, and vectors. Gene therapy involves introducing genes into cells to treat genetic defects or diseases. There are two main types - somatic gene therapy targets somatic cells to treat an individual without inheritance, while germline gene therapy targets germ cells to make the therapy heritable. Common strategies include gene augmentation, targeted killing of diseased cells, inhibition of gene expression, and correction of mutated genes. Methods include ex vivo therapy of cultured cells outside the body and in vivo therapy of direct gene transfer inside the body. Viral and non-viral vectors are used to deliver therapeutic genes to different target cells depending on the disease.
Gene therapy is a new tool used in combating different diseases. The majority of gene therapy clinical trials are focused on cancer and so it was no coincidence that the first commercial treatment in 2003 was for neoplasia. Currently there are a wide variety of gene therapy proposals involving a large number of anti tumour molecular mechanisms that will conceivably pave the way for highly effective a treatment options. Despite the significant advances that how been made in gene therapy in the fight against cancer, its efficacy,safety and commercial availability are still limited. Ms. Chetana D. Patil | Ms. Siddhi Chavan | Mr. Ritesh Kadam "Gene Therapy for Cancer Treatment" Published in International Journal of Trend in Scientific Research and Development (ijtsrd), ISSN: 2456-6470, Volume-3 | Issue-5 , August 2019, URL: https://www.ijtsrd.com/papers/ijtsrd26537.pdfPaper URL: https://www.ijtsrd.com/pharmacy/biotechnology-/26537/gene-therapy-for-cancer-treatment/ms-chetana-d-patil
INTRODUCTION OF GENE THERAPY, HISTORY OF GENE THERAPY, Process of gene therapy, Methods of gene therapy, Ex vivo gene therapy , In Vivo Gene Therapy , Uses of gene therapy, Target sites for Gene Therapy , Vectors for gene therapy , Viral Vectors, Non Viral Vectors,
Gene therapy involves introducing DNA into a patient's cells to treat a disease. There are four main strategies of gene therapy: gene augmentation therapy which adds a functional gene; targeted killing of specific cells like cancer cells; targeted mutation correction; and targeted inhibition of gene expression. Gene therapy shows promise for diseases like cystic fibrosis, hemophilia, cancer and more. Challenges remain around safely delivering genes to the right cells and avoiding immune responses.
Gene therapy aims to treat cystic fibrosis, a genetic disorder caused by mutations in the CFTR gene. Early clinical trials involved delivering a healthy copy of the CFTR gene to the lungs via viral vectors like adenovirus or adeno-associated virus, or non-viral liposomes. While these methods showed some promise, challenges remained in achieving adequate delivery, expression of the gene, and avoiding immune responses. Ongoing research focuses on improving gene transfer methods to make gene therapy more effective for cystic fibrosis.
A good comprehensive review of gene delivery and gene therapy. especially for master of pharmacy 2nd-semester students as per the PCI syllabus of subject Molecular pharmaceutics.
List of contents under this ppt :
{A} GENE THERAPY
(1) Definition
(2) Introduction
(3) History
(4) Ex-Vivo gene therapy
(5) In-Vivo gene therapy
(6) Germline gene therapy
(7) Advantages of gene therapy
(8) Disadvantages of gene therapy
(9) Potential target diseases for gene therapy
a. inherited disorders :- ADA SCID, Chronic granulomatous, Hemophelia
b. Cancer
{B} GENE DELIVERY
(1) Definition
(2) Introduction
(3) Types of vectors
a. Viral :- Retrovirus, Adenovirus, Adeno associated virus, Herps simplex virus
b. Non viral :-
Physical methods - Gene gun, Microinjection, Electroporation, Sonoporation
Chemical methods - Oligonucleotides, Lipoplexes, Polyplexes, Dendrimers, Nanoparticles.
Gene therapy - current status and future perspective Aleena Haqqi
The document discusses gene therapy, including its types, steps involved, applications, current status, limitations, and future perspective. It provides details on gene therapy techniques like gene augmentation and gene inhibition. Key points covered include current clinical trials focusing on cancer and monogenic diseases, the use of viral vectors, and challenges relating to funding, ethics, and ensuring safe and effective minimum dosages. The future of gene therapy is seen as promising for treating many currently incurable conditions if optimization and accessibility issues can be addressed.
This document discusses gene therapy, including its introduction, types, strategies, approaches, methods, target cells, and vectors. Gene therapy involves introducing genes into cells to treat genetic defects or diseases. There are two main types - somatic gene therapy targets somatic cells to treat an individual without inheritance, while germline gene therapy targets germ cells to make the therapy heritable. Common strategies include gene augmentation, targeted killing of diseased cells, inhibition of gene expression, and correction of mutated genes. Methods include ex vivo therapy of cultured cells outside the body and in vivo therapy of direct gene transfer inside the body. Viral and non-viral vectors are used to deliver therapeutic genes to different target cells depending on the disease.
Gene therapy is a new tool used in combating different diseases. The majority of gene therapy clinical trials are focused on cancer and so it was no coincidence that the first commercial treatment in 2003 was for neoplasia. Currently there are a wide variety of gene therapy proposals involving a large number of anti tumour molecular mechanisms that will conceivably pave the way for highly effective a treatment options. Despite the significant advances that how been made in gene therapy in the fight against cancer, its efficacy,safety and commercial availability are still limited. Ms. Chetana D. Patil | Ms. Siddhi Chavan | Mr. Ritesh Kadam "Gene Therapy for Cancer Treatment" Published in International Journal of Trend in Scientific Research and Development (ijtsrd), ISSN: 2456-6470, Volume-3 | Issue-5 , August 2019, URL: https://www.ijtsrd.com/papers/ijtsrd26537.pdfPaper URL: https://www.ijtsrd.com/pharmacy/biotechnology-/26537/gene-therapy-for-cancer-treatment/ms-chetana-d-patil
INTRODUCTION OF GENE THERAPY, HISTORY OF GENE THERAPY, Process of gene therapy, Methods of gene therapy, Ex vivo gene therapy , In Vivo Gene Therapy , Uses of gene therapy, Target sites for Gene Therapy , Vectors for gene therapy , Viral Vectors, Non Viral Vectors,
Gene therapy involves introducing DNA into a patient's cells to treat a disease. There are four main strategies of gene therapy: gene augmentation therapy which adds a functional gene; targeted killing of specific cells like cancer cells; targeted mutation correction; and targeted inhibition of gene expression. Gene therapy shows promise for diseases like cystic fibrosis, hemophilia, cancer and more. Challenges remain around safely delivering genes to the right cells and avoiding immune responses.
Gene therapy aims to treat cystic fibrosis, a genetic disorder caused by mutations in the CFTR gene. Early clinical trials involved delivering a healthy copy of the CFTR gene to the lungs via viral vectors like adenovirus or adeno-associated virus, or non-viral liposomes. While these methods showed some promise, challenges remained in achieving adequate delivery, expression of the gene, and avoiding immune responses. Ongoing research focuses on improving gene transfer methods to make gene therapy more effective for cystic fibrosis.
A good comprehensive review of gene delivery and gene therapy. especially for master of pharmacy 2nd-semester students as per the PCI syllabus of subject Molecular pharmaceutics.
List of contents under this ppt :
{A} GENE THERAPY
(1) Definition
(2) Introduction
(3) History
(4) Ex-Vivo gene therapy
(5) In-Vivo gene therapy
(6) Germline gene therapy
(7) Advantages of gene therapy
(8) Disadvantages of gene therapy
(9) Potential target diseases for gene therapy
a. inherited disorders :- ADA SCID, Chronic granulomatous, Hemophelia
b. Cancer
{B} GENE DELIVERY
(1) Definition
(2) Introduction
(3) Types of vectors
a. Viral :- Retrovirus, Adenovirus, Adeno associated virus, Herps simplex virus
b. Non viral :-
Physical methods - Gene gun, Microinjection, Electroporation, Sonoporation
Chemical methods - Oligonucleotides, Lipoplexes, Polyplexes, Dendrimers, Nanoparticles.
Gene therapy - current status and future perspective Aleena Haqqi
The document discusses gene therapy, including its types, steps involved, applications, current status, limitations, and future perspective. It provides details on gene therapy techniques like gene augmentation and gene inhibition. Key points covered include current clinical trials focusing on cancer and monogenic diseases, the use of viral vectors, and challenges relating to funding, ethics, and ensuring safe and effective minimum dosages. The future of gene therapy is seen as promising for treating many currently incurable conditions if optimization and accessibility issues can be addressed.
Gene therapy involves introducing genetic material into human cells to treat diseases. It aims to cure diseases by adding extra genes or replacing faulty genes. There are still many challenges to gene therapy, including developing safe and effective viral and non-viral vectors to deliver genes, avoiding unintended effects, and establishing regulations for clinical trials and production. As the field continues to advance, dedicated facilities and standardized procedures will be important to ensure patient safety.
Gene therapy involves introducing normal genes into cells to replace missing or defective genes and correct genetic disorders. The first gene therapy treatment occurred in 1990 for SCID. There are two main types - germline therapy would alter the germ cells and be heritable, while somatic therapy only affects the individual. Delivery of genes requires vectors like viruses that can transport the new gene into target cells. Challenges include developing vectors that don't cause immune responses, achieving long-term expression of the gene, and addressing disorders influenced by multiple genes. Risks include insertional mutagenesis and the development of tumors.
Gene therapy involves inserting normal genes into individuals to replace defective genes that cause disease. It has been used to treat various genetic diseases and cancers since the 1990s. While it offers promise for permanent treatment, gene therapy still faces challenges like short-term effects, immune responses, high costs, and difficulties with gene delivery methods that have limited its effectiveness so far. Continued research aims to overcome these obstacles.
Gene therapy involves transferring nucleic acids into cells to treat disease. The first approved gene therapy procedure was performed in 1990 on 4-year-old Ashanthi DeSilva who had severe combined immunodeficiency. Doctors removed her white blood cells, inserted the missing gene, and reinfused the cells. There are two main types of gene therapy: somatic and germline. Viral and non-viral vectors are used to deliver therapeutic genes. While gene therapy holds promise, safety issues remain due to potential short-lived effects, immune responses, and risks from viral vectors.
Gene therapy seeks to modify or manipulate genes to treat diseases. There are three main strategies for gene therapy: gene augmentation therapy which introduces a normal gene to compensate for an abnormal gene, targeted killing of specific cells, and targeted inhibition of gene expression. Gene therapy can be somatic, affecting most body cells, or germline, affecting eggs or sperm. Viral vectors are commonly used to deliver genes into cells, but non-viral methods also exist. Gene editing tools like ZFNs, TALENs, and CRISPR-Cas systems can also be used to precisely alter genes.
This document discusses gene therapy approaches including ex-vivo and in-vivo gene therapy. Ex-vivo gene therapy involves removing cells from the body, introducing therapeutic genes in culture, and reintroducing the modified cells. In-vivo gene therapy directly delivers therapeutic genes into target cells using viral or non-viral vectors. Examples of each approach are provided, such as using ex-vivo gene therapy to treat adenosine deaminase deficiency and in-vivo gene therapy to treat cystic fibrosis. In conclusion, while gene therapy has potential, it faces complexity and high costs, limiting current accessibility.
This document provides an overview of gene therapy. It defines key terms like genetics, genes, chromosomes, and therapy. It explains that gene therapy involves inserting a normal gene to replace an abnormal gene causing disease. Viruses and non-viral methods can be used as vectors to deliver therapeutic genes. While some gene therapies have shown promise for diseases like Parkinson's and immune deficiencies, the field has also faced challenges. Safety issues in early trials halted some research, but recent studies demonstrate progress in refining methods and potentially treating diseases like sickle cell anemia.
Gene therapy aims to alter the phenotype of diseased cells by introducing exogenous nucleic acids like DNA. It involves choosing a cellular target, effector mechanism, delivery method, and effector gene. For cancer, common targets are tumor cells themselves or normal cells like stem cells. Effector mechanisms include gene replacement, suicide genes, and RNA-directed strategies. Delivery can be viral, non-viral, or involve artificial virus-like particles. Effector genes, promoters, and suicide genes must be chosen to match the intended target and effect. Some gene therapy strategies have shown effectiveness in clinical trials for cancers like breast and prostate.
This document provides an overview of gene therapy, including its history, types, techniques, advantages, and disadvantages. Gene therapy is an experimental technique that aims to treat diseases by correcting defective genes or introducing new genes. It was first conceptualized in the 1960s but the first approved human gene therapy trial took place in 1990 for severe combined immunodeficiency. Current research focuses on diseases with no other cures like cancer, HIV, and genetic disorders. Gene transfer can be done through viral vectors like retroviruses or adenoviruses or non-viral methods. Potential advantages are a cure for inherited diseases, while disadvantages include risks of toxicity and unknown effects. Gene therapy remains an active area of research.
Gene therapy involves delivering therapeutic DNA into a patient's cells to treat disease. It was first conceptualized in 1972 and the first FDA-approved gene therapy for ADA-SCID occurred in 1990. Gene therapy targets diseases without other treatments, such as immune disorders, cystic fibrosis, and hemophilia. There are two main types - somatic gene therapy modifies body cells while germline modifies sperm or eggs. Viral and non-viral vectors are used to deliver genes in vivo or ex vivo gene therapy methods.
This presentation provides an overview of gene therapy. It introduces gene therapy as a technique for introducing genes into cells to prevent or cure diseases by correcting defective genes. The first approved gene therapy experiment occurred in 1990. There are two main types of gene therapy: somatic cell gene therapy, which transfers genes into somatic cells and is not inherited, and germline gene therapy, which transfers genes into germ cells and can be inherited. Gene delivery can be done in vivo or ex vivo using viral or non-viral vectors. While still facing challenges, gene therapy has potential to cure inherited diseases and possibly cancers and heart disease in the future.
Genetic disorders are caused by abnormalities in DNA, ranging from single gene mutations to full chromosome additions or deletions. Gene therapy aims to treat genetic disorders by inserting a normal gene to replace an abnormal one. Various methods can be used to deliver genes, including viral vectors like retroviruses. Some strategies include gene augmentation to restore lost function, targeted mutation correction, or inhibiting mutated gene expression. While offering hope for previously untreatable conditions, gene therapy carries risks like immune reactions and potential cancer formation that require further research.
Gene therapy involves delivering genes to correct genetic defects. It can augment or inhibit gene expression. Key steps include identifying the defective gene, cloning the normal gene, selecting a target cell/tissue, and inserting the gene. Genes can be delivered ex vivo by modifying cells outside the body or in vivo by direct gene transfer. Viral vectors like retroviruses are commonly used, but pose risks like activating proto-oncogenes. Gene therapy aims to treat genetic disorders and acquired diseases.
Gene therapy involves transferring therapeutic genes into diseased tissues to treat genetic disorders. The document discusses using gene therapy to treat hemophilia in dogs by infusing genes encoding coagulation factor IX via a one-hour procedure, resulting in gene expression for 15 months and blood clotting within 20 minutes. It also lists several diseases that may be treated with gene therapy and discusses general concerns with the approach, such as its short-lived nature and immune responses.
Gene therapy involves techniques that modify or manipulate genes to treat or prevent diseases. The first gene therapy treatment occurred in 1990 for severe combined immunodeficiency. There are four main approaches to gene therapy: inserting a normal gene to compensate for a defective one, replacing an abnormal gene with a normal one, repairing an abnormal gene, or altering gene regulation. Viruses are commonly used as vectors to deliver therapeutic genes into target cells, with retroviruses, adenoviruses, adeno-associated viruses, and herpes simplex viruses being some of the most widely used viral vectors, each with advantages and limitations.
Gene therapy involves introducing normal genes into cells containing defective genes to correct genetic disorders. There are four main approaches, including inserting a normal gene to compensate for a nonfunctional one. A viral vector delivers the therapeutic gene into a target cell, where it is inserted into the genome. Different delivery systems include using viruses like retroviruses, adenoviruses, adeno-associated viruses, and herpes simplex viruses. While gene therapy has been used to treat diseases like SCID, Parkinson's, and sickle cell disease, it faces limitations such as immune responses, risk of cancer from uncontrolled insertion, and high expenses.
This document provides an introduction to gene therapy. It defines gene therapy as a technique for correcting defective genes responsible for disease development by introducing one or more foreign genes into an organism. The document outlines the basic components of DNA, genes, and chromosomes. It then describes the different types and strategies of gene therapy, including replacement, knockout, suicide, and immunomodulatory gene therapy. The approaches, advantages, and problems of gene therapy are summarized.
Gene therapy involves inserting normal genes into cells to treat genetic diseases. The first successful gene therapy trial treated a patient with severe combined immunodeficiency (SCID) caused by adenosine deaminase (ADA) deficiency. The procedure involved removing the patient's lymphocytes, infecting them with a retrovirus carrying the normal ADA gene, allowing the gene to integrate and express ADA, and returning the modified lymphocytes to the patient to permanently produce the enzyme. This landmark trial established gene therapy as a viable treatment approach for genetic diseases.
This document provides an overview of pharmacoeconomics. It defines pharmacoeconomics as the application of economic analysis to pharmaceutical products and services, focusing on costs and outcomes. The document then discusses the history of pharmacoeconomics, perspectives and methodologies used, how it relates to drug development, and limitations. It emphasizes that pharmacoeconomics aims to optimize the value of pharmaceutical expenditures.
This document discusses Contract Research Organizations (CROs) and their role in supporting pharmaceutical clinical trials. It provides the following key points:
- CROs are service organizations that conduct clinical trials and other drug development work on behalf of pharmaceutical companies. This outsourcing has grown as drug development has become more complex.
- When deciding whether and how to use a CRO, companies consider tactical, maximal, or strategic outsourcing based on their internal resources. Selecting the right CRO requires evaluating their capabilities, compatibility with the sponsor's needs, and costs.
- Managing the sponsor-CRO relationship is critical to ensure success. This involves clearly defining roles and responsibilities, establishing performance metrics,
Gene therapy involves introducing genetic material into human cells to treat diseases. It aims to cure diseases by adding extra genes or replacing faulty genes. There are still many challenges to gene therapy, including developing safe and effective viral and non-viral vectors to deliver genes, avoiding unintended effects, and establishing regulations for clinical trials and production. As the field continues to advance, dedicated facilities and standardized procedures will be important to ensure patient safety.
Gene therapy involves introducing normal genes into cells to replace missing or defective genes and correct genetic disorders. The first gene therapy treatment occurred in 1990 for SCID. There are two main types - germline therapy would alter the germ cells and be heritable, while somatic therapy only affects the individual. Delivery of genes requires vectors like viruses that can transport the new gene into target cells. Challenges include developing vectors that don't cause immune responses, achieving long-term expression of the gene, and addressing disorders influenced by multiple genes. Risks include insertional mutagenesis and the development of tumors.
Gene therapy involves inserting normal genes into individuals to replace defective genes that cause disease. It has been used to treat various genetic diseases and cancers since the 1990s. While it offers promise for permanent treatment, gene therapy still faces challenges like short-term effects, immune responses, high costs, and difficulties with gene delivery methods that have limited its effectiveness so far. Continued research aims to overcome these obstacles.
Gene therapy involves transferring nucleic acids into cells to treat disease. The first approved gene therapy procedure was performed in 1990 on 4-year-old Ashanthi DeSilva who had severe combined immunodeficiency. Doctors removed her white blood cells, inserted the missing gene, and reinfused the cells. There are two main types of gene therapy: somatic and germline. Viral and non-viral vectors are used to deliver therapeutic genes. While gene therapy holds promise, safety issues remain due to potential short-lived effects, immune responses, and risks from viral vectors.
Gene therapy seeks to modify or manipulate genes to treat diseases. There are three main strategies for gene therapy: gene augmentation therapy which introduces a normal gene to compensate for an abnormal gene, targeted killing of specific cells, and targeted inhibition of gene expression. Gene therapy can be somatic, affecting most body cells, or germline, affecting eggs or sperm. Viral vectors are commonly used to deliver genes into cells, but non-viral methods also exist. Gene editing tools like ZFNs, TALENs, and CRISPR-Cas systems can also be used to precisely alter genes.
This document discusses gene therapy approaches including ex-vivo and in-vivo gene therapy. Ex-vivo gene therapy involves removing cells from the body, introducing therapeutic genes in culture, and reintroducing the modified cells. In-vivo gene therapy directly delivers therapeutic genes into target cells using viral or non-viral vectors. Examples of each approach are provided, such as using ex-vivo gene therapy to treat adenosine deaminase deficiency and in-vivo gene therapy to treat cystic fibrosis. In conclusion, while gene therapy has potential, it faces complexity and high costs, limiting current accessibility.
This document provides an overview of gene therapy. It defines key terms like genetics, genes, chromosomes, and therapy. It explains that gene therapy involves inserting a normal gene to replace an abnormal gene causing disease. Viruses and non-viral methods can be used as vectors to deliver therapeutic genes. While some gene therapies have shown promise for diseases like Parkinson's and immune deficiencies, the field has also faced challenges. Safety issues in early trials halted some research, but recent studies demonstrate progress in refining methods and potentially treating diseases like sickle cell anemia.
Gene therapy aims to alter the phenotype of diseased cells by introducing exogenous nucleic acids like DNA. It involves choosing a cellular target, effector mechanism, delivery method, and effector gene. For cancer, common targets are tumor cells themselves or normal cells like stem cells. Effector mechanisms include gene replacement, suicide genes, and RNA-directed strategies. Delivery can be viral, non-viral, or involve artificial virus-like particles. Effector genes, promoters, and suicide genes must be chosen to match the intended target and effect. Some gene therapy strategies have shown effectiveness in clinical trials for cancers like breast and prostate.
This document provides an overview of gene therapy, including its history, types, techniques, advantages, and disadvantages. Gene therapy is an experimental technique that aims to treat diseases by correcting defective genes or introducing new genes. It was first conceptualized in the 1960s but the first approved human gene therapy trial took place in 1990 for severe combined immunodeficiency. Current research focuses on diseases with no other cures like cancer, HIV, and genetic disorders. Gene transfer can be done through viral vectors like retroviruses or adenoviruses or non-viral methods. Potential advantages are a cure for inherited diseases, while disadvantages include risks of toxicity and unknown effects. Gene therapy remains an active area of research.
Gene therapy involves delivering therapeutic DNA into a patient's cells to treat disease. It was first conceptualized in 1972 and the first FDA-approved gene therapy for ADA-SCID occurred in 1990. Gene therapy targets diseases without other treatments, such as immune disorders, cystic fibrosis, and hemophilia. There are two main types - somatic gene therapy modifies body cells while germline modifies sperm or eggs. Viral and non-viral vectors are used to deliver genes in vivo or ex vivo gene therapy methods.
This presentation provides an overview of gene therapy. It introduces gene therapy as a technique for introducing genes into cells to prevent or cure diseases by correcting defective genes. The first approved gene therapy experiment occurred in 1990. There are two main types of gene therapy: somatic cell gene therapy, which transfers genes into somatic cells and is not inherited, and germline gene therapy, which transfers genes into germ cells and can be inherited. Gene delivery can be done in vivo or ex vivo using viral or non-viral vectors. While still facing challenges, gene therapy has potential to cure inherited diseases and possibly cancers and heart disease in the future.
Genetic disorders are caused by abnormalities in DNA, ranging from single gene mutations to full chromosome additions or deletions. Gene therapy aims to treat genetic disorders by inserting a normal gene to replace an abnormal one. Various methods can be used to deliver genes, including viral vectors like retroviruses. Some strategies include gene augmentation to restore lost function, targeted mutation correction, or inhibiting mutated gene expression. While offering hope for previously untreatable conditions, gene therapy carries risks like immune reactions and potential cancer formation that require further research.
Gene therapy involves delivering genes to correct genetic defects. It can augment or inhibit gene expression. Key steps include identifying the defective gene, cloning the normal gene, selecting a target cell/tissue, and inserting the gene. Genes can be delivered ex vivo by modifying cells outside the body or in vivo by direct gene transfer. Viral vectors like retroviruses are commonly used, but pose risks like activating proto-oncogenes. Gene therapy aims to treat genetic disorders and acquired diseases.
Gene therapy involves transferring therapeutic genes into diseased tissues to treat genetic disorders. The document discusses using gene therapy to treat hemophilia in dogs by infusing genes encoding coagulation factor IX via a one-hour procedure, resulting in gene expression for 15 months and blood clotting within 20 minutes. It also lists several diseases that may be treated with gene therapy and discusses general concerns with the approach, such as its short-lived nature and immune responses.
Gene therapy involves techniques that modify or manipulate genes to treat or prevent diseases. The first gene therapy treatment occurred in 1990 for severe combined immunodeficiency. There are four main approaches to gene therapy: inserting a normal gene to compensate for a defective one, replacing an abnormal gene with a normal one, repairing an abnormal gene, or altering gene regulation. Viruses are commonly used as vectors to deliver therapeutic genes into target cells, with retroviruses, adenoviruses, adeno-associated viruses, and herpes simplex viruses being some of the most widely used viral vectors, each with advantages and limitations.
Gene therapy involves introducing normal genes into cells containing defective genes to correct genetic disorders. There are four main approaches, including inserting a normal gene to compensate for a nonfunctional one. A viral vector delivers the therapeutic gene into a target cell, where it is inserted into the genome. Different delivery systems include using viruses like retroviruses, adenoviruses, adeno-associated viruses, and herpes simplex viruses. While gene therapy has been used to treat diseases like SCID, Parkinson's, and sickle cell disease, it faces limitations such as immune responses, risk of cancer from uncontrolled insertion, and high expenses.
This document provides an introduction to gene therapy. It defines gene therapy as a technique for correcting defective genes responsible for disease development by introducing one or more foreign genes into an organism. The document outlines the basic components of DNA, genes, and chromosomes. It then describes the different types and strategies of gene therapy, including replacement, knockout, suicide, and immunomodulatory gene therapy. The approaches, advantages, and problems of gene therapy are summarized.
Gene therapy involves inserting normal genes into cells to treat genetic diseases. The first successful gene therapy trial treated a patient with severe combined immunodeficiency (SCID) caused by adenosine deaminase (ADA) deficiency. The procedure involved removing the patient's lymphocytes, infecting them with a retrovirus carrying the normal ADA gene, allowing the gene to integrate and express ADA, and returning the modified lymphocytes to the patient to permanently produce the enzyme. This landmark trial established gene therapy as a viable treatment approach for genetic diseases.
This document provides an overview of pharmacoeconomics. It defines pharmacoeconomics as the application of economic analysis to pharmaceutical products and services, focusing on costs and outcomes. The document then discusses the history of pharmacoeconomics, perspectives and methodologies used, how it relates to drug development, and limitations. It emphasizes that pharmacoeconomics aims to optimize the value of pharmaceutical expenditures.
This document discusses Contract Research Organizations (CROs) and their role in supporting pharmaceutical clinical trials. It provides the following key points:
- CROs are service organizations that conduct clinical trials and other drug development work on behalf of pharmaceutical companies. This outsourcing has grown as drug development has become more complex.
- When deciding whether and how to use a CRO, companies consider tactical, maximal, or strategic outsourcing based on their internal resources. Selecting the right CRO requires evaluating their capabilities, compatibility with the sponsor's needs, and costs.
- Managing the sponsor-CRO relationship is critical to ensure success. This involves clearly defining roles and responsibilities, establishing performance metrics,
This document discusses multiple drug resistance (MDR) and the role of ATP-binding cassette (ABC) transporters. ABC transporters are membrane proteins that transport molecules across cell membranes using ATP hydrolysis. They are involved in MDR by pumping drugs out of cells. The document describes the structure and mechanisms of both importers and exporters. Importers import nutrients into cells using binding proteins, while exporters export toxins using conformational changes driven by ATP binding and hydrolysis. Understanding ABC transporters may help overcome MDR in cancer and bacterial infections.
The document summarizes cystic fibrosis, including:
1) It is caused by mutations in the CFTR gene which encodes a chloride channel protein, leading to thick mucus in organs like the lungs and pancreas.
2) The most common mutation is DeltaF508. Symptoms include salty skin, lung infections, and poor growth.
3) Treatments aim to clear mucus from the lungs and control infections, while enzymes and vitamins address digestive issues. Gene therapy aims to replace the defective CFTR gene.
Pharmacoepidemiology is the study of the use and effects of drugs in large populations. It combines the fields of clinical pharmacology and epidemiology. Recent data shows that adverse drug reactions cause 100,000 deaths and 1.5 million hospitalizations in the US each year, yet 20-70% may be preventable. Pharmacoepidemiology aims to detect adverse drug reactions early through observational studies in order to educate healthcare providers and the public about safer medication use. Key study types include case series, case-control studies, cohort studies, cross-sectional studies, and experimental studies. Drug utilization studies also fall under pharmacoepidemiology and evaluate factors related to prescribing, dispensing, administering, and taking
Pharmacovigilance involves monitoring approved drugs to detect adverse effects, assess risks, and prevent harm. It aims to improve patient safety by identifying unknown risks from drugs and informing regulatory decisions. Various methods are used, including spontaneous reporting of adverse drug reactions, active surveillance, and observational studies. Stringent pharmacovigilance is important given historical examples of drugs that caused significant harm after approval, demonstrating the need for ongoing monitoring of drug safety.
This document discusses Contract Research Organizations (CROs) and their role in supporting the pharmaceutical industry. It provides information on:
- What CROs are and the services they offer pharmaceutical companies, such as conducting clinical trials.
- Best practices for pharmaceutical companies when partnering with CROs, including clearly defining study specifications, selecting the right CRO based on capability and cost, and managing the relationship.
- Key aspects of contracts between sponsors and CROs such as defining roles and responsibilities, compliance with regulations, intellectual property, and termination policies.
The document aims to provide guidance to pharmaceutical companies on effectively partnering with CROs for outsourced drug development activities.
Pharmacovigilance involves monitoring approved drugs to detect adverse effects, assess risks, prevent harm and promote safe use. It aims to improve public health by identifying unknown risks from case reports and studies. Several methods are used including spontaneous reporting, active surveillance and observational studies. Organizations like WHO and regulatory authorities play important roles in pharmacovigilance. The goal is continual assessment of benefit-risk profiles to optimize treatment outcomes.
Cell lines are immortal cell cultures that are used extensively in biomedical research. Some key uses of cell lines include modeling disease and testing drug toxicity. Common cell lines include HeLa cells, MCF-7 breast cancer cells, and VERO monkey kidney cells. Cell lines are also used to produce biological products like vaccines, antibodies, and recombinant proteins. Respiratory cell lines help study lung diseases while pancreatic beta cell lines aid diabetes research. The duck cell line EB66 is being used commercially for viral vaccine production.
This document summarizes mechanisms of multidrug resistance (MDR) in organisms like bacteria and cancer cells. It discusses how MDR occurs through several mechanisms including enzymatic degradation of drugs, mutation of drug binding sites, downregulation of membrane proteins, and increased activity of efflux pumps that export drugs from cells. A major contributor to MDR is the increased expression of ATP-binding cassette (ABC) transporters like P-glycoprotein, which use ATP hydrolysis to actively transport various drugs out of cells, reducing their effectiveness. Understanding the structure and transport mechanisms of ABC transporters may help in developing new strategies to overcome MDR.
Monoclonal antibodies (mAbs) have various applications including diagnostic, therapeutic, and catalytic uses. Diagnostically, mAbs are used in pregnancy tests, disease diagnostics, and immunohistochemistry. Therapeutically, mAbs treat cancer, transplant rejection, and autoimmune diseases by mechanisms like enhancing immune response, blocking growth signals, inhibiting angiogenesis or cytokines. Some FDA-approved mAbs for cancer include rituximab, trastuzumab, and bevacizumab. Conjugated mAbs can deliver toxins or radiation directly to tumors. mAbs also have applications in areas like organ transplantation, autoimmune diseases, and drug targeting.
The document discusses programmed cell death or apoptosis. It begins by defining apoptosis as a regulated process where cells self-degrade to eliminate unwanted or damaged cells. Between 50-70 billion cells die daily in humans through apoptosis. The document then covers the history of apoptosis research and discovery. It discusses the role of caspases as executioners of apoptosis and the intrinsic and extrinsic pathways. Conditions where apoptosis is increased or decreased are examined, along with potential therapeutic targets like caspase inhibitors.
The document discusses the biology of vascular endothelium. It states that the endothelium is a thin layer of cells that lines the interior surface of blood vessels and lymphatic vessels, forming an interface between circulating blood/lymph and the vessel wall. The basic constituents of blood vessel walls are endothelial cells, smooth muscle cells, extracellular matrix, elastin, and collagen. The endothelium regulates vascular tone by releasing both vasodilators like nitric oxide, prostacyclin, and endothelium-derived hyperpolarizing factor and vasoconstrictors like endothelin-1 and thromboxane A2. Endothelial dysfunction, an imbalance between these factors, contributes to conditions like hypertension, atherosclerosis, and diabetes.
This document provides an overview of pharmacoeconomics. It defines pharmacoeconomics as the application of economic analysis to pharmaceutical products and services, focusing on costs and outcomes. The document then covers:
- The history and perspectives of pharmacoeconomics
- Common methodologies like cost-benefit analysis, cost-effectiveness analysis, and cost-utility analysis
- How pharmacoeconomics can be applied at different stages of drug development and to support pricing and reimbursement decisions
- The relationships between pharmacoeconomics, outcomes research, and pharmaceutical care
In 3 sentences or less, this document summarizes key concepts in pharmacoeconomics like its definition, common methodologies, and applications in drug
Cell adhesion molecules and matrix proteinsUSmile Ï Ṩṃïlệ
Cell adhesion molecules are proteins located on cell surfaces that are involved in binding between cells or between cells and the extracellular matrix. The three main types are cadherins, integrins, and selectins. Cadherins are calcium-dependent proteins that mediate cell-cell adhesion. Integrins are transmembrane receptors that bind to components of the extracellular matrix and mediate cell-matrix adhesion as well as cell signaling. Selectins mediate the initial capture and rolling of leukocytes along vascular surfaces. Cell adhesion molecules play important physiological roles in processes like leukocyte trafficking, blood coagulation, and morphogenesis. They also have applications as therapeutic targets in areas such as cancer, osteoporosis, and inflammatory diseases.
Cytokines are low molecular weight polypeptides or glycoproteins that are secreted by cells and have various functions including mediating and regulating immune responses and inflammatory reactions. Cytokines are produced by lymphocytes, monocytes, macrophages, mast cells, glial cells and other cells. They act through autocrine, paracrine or endocrine mechanisms and initiate their actions by binding to specific membrane receptors. Cytokines have pleiotropic, redundant, synergistic and antagonistic effects and form a cytokine network. The major classes of cytokines include interleukins, tumor necrosis factors, interferons, colony stimulating factors, transforming growth factors and chemokines. Cytokines play important roles in various diseases and their therapeutic uses include treatment
The document discusses the concept of essential medicines and rational use of drugs. It defines essential medicines as those that meet the priority health care needs of the population. The WHO publishes a Model List of Essential Medicines every two years to guide countries in developing their own national lists. Educational, managerial, economic and regulatory strategies can be used to promote rational drug use and selection of cost-effective treatments. Pharmacists can play a role through drug selection, inventory control, patient education, and pharmaceutical care.
This document discusses multiple drug resistance (MDR) in several organisms and contexts. It begins with an introduction to MDR and its mechanisms, including enzymatic degradation, mutations, efflux pumps, and decreased membrane permeability. Specific examples of MDR are then explored in tuberculosis, bacteria, cancer cells, HIV, and malaria. The mechanisms of resistance and genes involved vary by organism but often involve efflux pump proteins like P-glycoprotein. MDR is an increasing public health issue due to its role in antibiotic resistance.
This document provides information about gene therapy. It discusses that gene therapy involves genetically modifying patient cells to treat or alleviate disease. There are two main types - ex vivo therapy where cells are modified outside the body and transplanted back in, and in vivo therapy where genetic material is directly transferred into patient cells. Viruses like retroviruses, adenoviruses, and lentiviruses are often used as vectors to deliver genetic material due to their ability to efficiently transfer genes. The document outlines different applications of gene therapy and discusses strategies used depending on disease characteristics.
In this slide, You will get to learn abut Gene Therapy and different types of gene therapy. Various method of Gene Therapy and Advantage & Disadvantage and Recent advances in Gene Therapy.
This document discusses ex vivo gene therapy methods. It involves 3 main steps:
1) Isolation of cells from a patient with a genetic defect
2) Culturing and genetically modifying the cells to correct the defect
3) Transplanting the modified cells back into the patient. As the patient's own cells are used, there is no risk of immune rejection. Viral and non-viral vectors are used to deliver therapeutic genes to the cultured cells. Ex vivo gene therapy is applicable to tissues that can be removed, modified and reintroduced into the body.
Gene therapy is an experimental treatment that involves introducing genetic material into a person’s cells to fight or prevent disease. Researchers are studying gene therapy for a number of diseases, such as severe combined immuno-deficiencies, hemophilia, Parkinson's disease, cancer and even HIV, through a number of different approaches (see video: 'Gene Therapy a new tool to cure human diseases'). A gene can be delivered to a cell using a carrier known as a “vector.” The most common types of vectors used in gene therapy are viruses. The viruses used in gene therapy are altered to make them safe, although some risks still exist with gene therapy. The technology is still in its infancy, but it has been used with some success.
Gene therapy involves introducing genes into cells to treat or cure diseases. It works by correcting defective genes that cause illnesses. The first approved human gene therapy trial took place in 1990 in the US and treated a patient with ADA-SCID. There are two main types of gene therapy - somatic cell gene therapy, which treats genes in body cells but is not inherited, and germline gene therapy, which treats genes in reproductive cells and can be passed to offspring. Gene therapy approaches include in vivo delivery of genes directly into tissues and ex vivo therapy involving culturing and modifying cells outside the body before reinsertion. Viral and non-viral vectors are used to transport therapeutic genes into target cells.
Gene therapy involves introducing normal genes into patients to compensate for mutated genes that cause disease. The first gene therapy trial treated a girl with severe combined immunodeficiency. While it initially strengthened her immune system, the effects only lasted a few months. Gene therapy shows promise for diseases caused by single gene defects like cystic fibrosis, but faces challenges like short-lived effects, immune responses, and safety issues. Continued research aims to address these challenges through techniques like RNA interference and improved gene delivery methods.
NUCLEIC ACID BASED THERAPEUTIC DELIVERY SYSTEM by pramesh..pptxPRAMESHPANWAR1
Name of the title: Nucleic Acid-Based Therapeutic Delivery System.
It includes information about nucleic acid, gene therapy, and its type, a method to deliver the desired DNA, i.e., vectors and their types, with proper examples and diagrams, and how these things help in delivering a nucleic acid-based therapeutic drug delivery system.
The document discusses gene therapy as a potential treatment for various diseases. It describes how gene therapy can be used to treat diseases by gene transplantation, correction, or augmentation. The two main approaches to gene therapy are in vivo, where genetic material is directly transferred into a patient's body, and ex vivo, where genetic material is first transferred into cells grown in vitro and then returned to the patient. Specific diseases that may be treatable with gene therapy include severe combined immunodeficiency, hemophilia, cystic fibrosis, cancer, and various neurological and infectious diseases. Cystic fibrosis is discussed in more detail as the most common lethal genetic disorder in Caucasian populations.
Gene therapy involves introducing normal genes into patients to replace abnormal genes that cause disease. Viruses are often used as vectors to deliver therapeutic genes to target cells. While promising for treating genetic disorders, gene therapy faces challenges like short-lived effects, immune responses, and difficulties targeting multi-gene disorders. It also raises ethical issues around heritable genetic modifications and access to expensive new treatments. Continued research aims to address these scientific and ethical considerations to realize gene therapy's potential.
Gene therapy involves introducing genetic material into cells to treat or prevent disease. It has the potential to cure genetic disorders by correcting the underlying genetic defect. There are two main types - somatic gene therapy, which affects only targeted cells and is safer, and germline gene therapy which can permanently alter the genes and be passed to offspring. Recent advances include FDA-approved CAR-T immunotherapies for cancer and the first gene therapy approved for an inherited retinal disease. Challenges remain regarding delivery methods, safety, and ethical issues.
This presentation focuses on the science of Gene Therapy, the techniques of germ-line and somatic gene therapy and the mechanism of curing diseases and disorders using gene therapy. The presentation starts by discussing some common basic terms from genetics and moves on to the historical development of gene therapy techniques in chronological order. The different types of gene therapy techniques and their mechanisms have been discussed in detail subsequently. In concluding slides, some commercially available gene therapy products are mentioned and challenges of gene-therapy techniques have been highlighted.
Nucleic acid and cell based therapies involve gene therapy and cell therapy. Gene therapy aims to introduce new genes into cells to treat genetic diseases by replacing defective genes. Early gene therapy trials focused on ex vivo and in vivo approaches. Viral vectors like retroviruses were primarily used but posed safety risks. Non-viral methods using naked DNA or vectors like liposomes were developed. Diseases targeted include cancer, genetic disorders, and AIDS. Antisense oligonucleotides can bind mRNA and inhibit gene expression. RNA interference uses short interfering RNAs to induce sequence-specific gene silencing. Ribozymes and aptamers also provide nucleic acid based therapeutic approaches.
Gene therapy involves modifying genes to treat or cure disease. It works by replacing mutated genes, inactivating abnormal genes, or introducing new genes. Early successes treated immune deficiencies, but challenges remain in achieving long-term effects without side effects. Promising areas are treating inherited retinal diseases and Parkinson's through localized delivery of therapeutic genes using viral or non-viral vectors. While offering potential cures, gene therapy also raises ethical issues that require ongoing discussion.
This document discusses gene therapy, including its strategies, methods of delivery, history, and applications. It provides an overview of key concepts such as:
1. Gene therapy aims to treat genetic diseases by inserting normal genes into cells to compensate for abnormal genes. Strategies include gene replacement, gene augmentation, and gene inhibition.
2. Viruses are commonly used as vectors to deliver therapeutic genes. Retroviruses and adenoviruses integrate into the genome but can cause mutations, while adenoviruses are safer but less efficient.
3. The basic process involves isolating the normal gene, inserting it into a viral vector, infecting target cells, and having the cells produce functional proteins to return to
Gene therapy refers to the insertion of genetic material to correct a genetic defect.
In gene therapy, a "normal" gene is inserted into the genome to replace an "abnormal," disease-causing gene
Gene therapy is an approach to treating diseases by modifying or correcting abnormal genes. It can involve replacing a mutated gene, inactivating an abnormal gene, or introducing a new gene. While the concept was introduced in the 1960s, the first approved human gene therapy trial took place in 1990. Since then, gene therapy has successfully treated various diseases like blindness, immune deficiencies, and Parkinson's disease symptoms. Viral and non-viral vectors are used to deliver therapeutic genes, with both methods having advantages and disadvantages. Ongoing research continues to expand the diseases that can be treated with this promising approach.
Gene therapy types advantags disadvantagesSUJITHA MARY
Gene therapy involves inserting normal genes into patients to replace abnormal genes responsible for disease. There are two main types of gene therapy: ex vivo involves removing cells, modifying them, and reinserting them; in vivo delivers genes directly into target tissues. Viral and non-viral vectors are used to deliver genes. While gene therapy has potential to treat many diseases, it also has disadvantages like short-term effects and safety risks that must still be addressed.
Gene therapy involves introducing genes into cells to treat or prevent disease. It works by correcting defective genes that cause disease or by making cells produce products to treat the disease. The first approved gene therapy treated a girl for ADA-SCID. There are two main approaches - in vivo therapy directly delivers genes into body cells, while ex vivo therapy transfers genes to cultured cells before reinsertion. Viral vectors like retroviruses and adenoviruses are often used due to their ability to deliver genes, but come with risks like insertional mutagenesis. Non-viral methods include physical methods like microinjection and chemical methods using liposomes. Gene therapy shows promise for diseases like cancer, cardiovascular disease, and neurological disorders.
Stem cell therapy involves using stem cells to treat diseases. There are several types of stem cells including embryonic stem cells derived from embryos, adult stem cells found in tissues, and induced pluripotent stem cells created from adult cells. Stem cell therapy works by replacing damaged cells with healthy stem cell-derived cells. It is being used and researched for treating conditions like cancer, diabetes, heart disease, and neurological disorders. However, the use of embryonic stem cells raises some ethical issues as it involves the destruction of embryos.
Cystic fibrosis is caused by mutations in the CFTR gene which encodes a chloride channel protein. Defective CFTR proteins result in thick mucus in the lungs, pancreas and other organs leading to infections and impaired digestion. The disease is diagnosed via sweat tests or genetic testing. Treatment focuses on clearing mucus from the lungs and controlling infections with antibiotics and mucus-thinning drugs. Gene therapy aims to replace the defective CFTR gene.
1. Chirality refers to molecular compounds that are non-superimposable on their mirror images and thus exist as two enantiomers. Recognition of chirality in compounds is important in pharmacology.
2. Many drugs are chiral, and their enantiomers may have different pharmacological effects ranging from no activity to distinct activities. This is due to biological molecules like receptors being chiral.
3. There has been a shift in drug development from racemic drug mixtures to single enantiomers due to issues like thalidomide and differences in enantiomer activity and side effects. Many current drugs have been redeveloped as single enantiomers.
This document discusses nutraceuticals and their health benefits. It defines nutraceuticals as foods or food components that provide medical or health benefits, including the prevention and treatment of disease. Some key points include:
- Nutraceuticals include probiotics, prebiotics, antioxidants, herbs, and other functional food components. They are associated with preventing major diseases like heart disease, cancer, hypertension and diabetes.
- Nutraceuticals are preferred over pharmaceuticals due to being natural, less toxic substances that consumers believe are safer. They are also seen as helping to reduce healthcare costs.
- The document outlines various categories of nutraceuticals and provides examples of components and their health functions
Gene therapy involves introducing genetic material into cells to treat disease. It is used for inherited disorders, cancers, and infectious diseases. Viruses are commonly used as vectors to deliver therapeutic genes, though safety issues exist. Some early successes included treating severe combined immunodeficiency using retroviruses to deliver the ADA gene ex vivo. However, the first death in gene therapy occurred treating ornithine transcarbamylase deficiency using an adenovirus vector, highlighting the risks. Improved delivery methods like liposomes may help overcome current limitations and advance this promising approach.
Cell lines are immortal cell cultures that are used extensively in biomedical research. Some key uses of cell lines include studying basic cell biology, testing drug toxicity, cancer research, virology studies, and gene therapy. Common cell lines include HeLa cells from cervical cancer tissue and MCF-7 cells from breast cancer tissue. Cell lines are also important in the production of vaccines, antibodies, and other biological products through recombinant protein expression in cultured cells. Respiratory and pancreatic beta cell lines have applications in research on inflammation, diabetes, and other diseases.
Chemokines are small proteins that direct the movement of white blood cells to sites of injury or infection. They are classified based on structural characteristics like the positioning of conserved cysteine residues. The four main classes are CC, CXC, C, and CX3C chemokines. Chemokines bind to G protein-coupled receptors on cells and signal through G proteins and secondary messengers to induce cell migration. Chemokines play roles in processes like inflammation, immunity, and cancer and are implicated in diseases like HIV, arthritis, and transplant rejection.
TEST BANK For Community Health Nursing A Canadian Perspective, 5th Edition by...Donc Test
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Basavarajeeyam is a Sreshta Sangraha grantha (Compiled book ), written by Neelkanta kotturu Basavaraja Virachita. It contains 25 Prakaranas, First 24 Chapters related to Rogas& 25th to Rasadravyas.
- Video recording of this lecture in English language: https://youtu.be/kqbnxVAZs-0
- Video recording of this lecture in Arabic language: https://youtu.be/SINlygW1Mpc
- Link to download the book free: https://nephrotube.blogspot.com/p/nephrotube-nephrology-books.html
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Promoting Wellbeing - Applied Social Psychology - Psychology SuperNotesPsychoTech Services
A proprietary approach developed by bringing together the best of learning theories from Psychology, design principles from the world of visualization, and pedagogical methods from over a decade of training experience, that enables you to: Learn better, faster!
2. What is gene therapy? Why is it used?
• Gene therapy is the application of genetic principles in
the treatment of human disease
• Gene therapy = Introduction of genetic material into
normal cells in order to:
– counteract the effect of a disease gene or
– introduce a new function
• GT is used to correct a deficient phenotype so that
sufficient amounts of a normal gene product are
synthesized to improve a genetic disorder
• Can be applied as therapy for cancers, inherited
disorders, infectious diseases, immune system
disorders
4. History of gene therapy
1930’s “genetic engineering” - plant/animal breeding
60’s first ideas of using genes therapeutically
50’s-70’s gene transfer developed
70’s-80’s recombinant DNA technology
1990 first GT in humans (ADA deficiency)
2001 596 GT clinical trials (3464 patients)
5. Human Genome Project
• A genome is all the DNA in an organism,
including its genes. Genes carry information for
making all the proteins required by all organisms
• Variations in structure of person’s genes
collectively helps define us as individuals
• Rationale for GT is based on knowledge of the
human genetic code
• Began formally in 1990, set as a 13 year project.
6. HGP GOALS
• Identify aproximately 30,000 genes
• Determine 3 billion chemical base pairs that
make up human DNA
• Store information in databases
• Improve tools for data analysis
• Address ethical, legal and social issues that
might arise from this
7. HGP cont…
• Achieve these goals,
researchers look at
genetic makeup of other
organisms
• This project is important
because the government's
dedication to the transfer
of technology to the
private sector
8. Three types of gene therapy:
• Monogenic gene therapy
• Provides genes to encode for the production of a
specific protein
• Cystic fibrosis, Muscular dystrophy, Sickle cell
disease, Haemophilia, SCID
• Suicide gene therapy
• Provide ‘suicide’ genes to target cancer cells for
destruction
• Cancer
• Antisense gene therapy
• Provides a single stranded gene in an’antisense’
(backward) orientation to block the production of
harmful proteins
• AIDS/HIV
9. Different Delivery Systems are
Available
• In vivo versus ex vivo
– In vivo = delivery of genes takes place in the body
– Ex vivo = delivery takes place out of the body, and
then cells are placed back into the body
10. Getting genes into cells
• In vivo versus ex vivo
– In vivo = intravenous or intramuscular or non-
invasive (‘sniffable’)
– Ex vivo = hepatocytes, skin fibroblasts,
haematopoietic cells (‘bioreactors’)
• Gene delivery approaches
– Physical methods
– Non-viral vectors
– Viral vectors
11. • In vivo techniques usually utilize viral vectors
– Virus = carrier of desired gene
– Virus is usually “crippled” to disable its ability to cause disease
– Viral methods have proved to be the most efficient to date
– Many viral vectors can stable integrate the desired gene into
the target cell’s genome
In vivo techniques
– Problem: Replication defective viruses adversely
affect the virus’ normal ability to spread genes in
the body
• Reliant on diffusion and spread
• Hampered by small intercellular spaces for transport
• Restricted by viral-binding ligands on cell surface
therefore cannot advance far.
12. “Viruses are highly evolved
natural vectors
for the transfer of foreign genetic information
into cells”
But to improve safety,
they need to be replication defective
Viral vectors
13. Compared to naked
DNA, virus particles
provide a relatively
efficient means of
transporting DNA into
cells, for expression in
the nucleus as
recombinant genes
(example =
adenovirus).
Viral vectors
16. Delivery System of Choice = Viral
Vectors
A. Rendering virus vector harmless
Remove harmful genes “cripple” the virus
Example – removal of env gene virus is not capable
of producing a functional envelope
Vectors needed in very large numbers to achieve
successful delivery of new genes into patient’s cells
Vectors must be propagated in large numbers in cell
culture (109
) with the aid of a helper virus
18. Advantages
• High transduction efficiency
• Insert size up to 8kbHigh viral titer (1010
-1013
)
• Infects both replicating and differentiated cells
Disadvantages
• Expression is transient (viral DNA does not integrate)
• Viral proteins can be expressed in host following vector
administration
• In vivo delivery hampered by host immune response
Adenovirus
19. Advantages
• Large insert size
• Could provide long- term CNS gene expression
• High titer
Disadvantages
• System currently under development
• Current vectors provide transient expression
• Low transduction efficiency
Herpes Simplex Virus
20. • Ex vivo manipulation techniques
– Electroporation
– Liposomes
– Calcium phosphate
– Gold bullets (fired within helium pressurized gun)
– Retrotransposons (jumping genes – early days)
– Human artificial chromosomes
Ex vivo
23. • In aqueous solution, polar phospholipids form ordered
aggregates to minimize hydrophobic interactions
• Lipid shape and conditions of formation affect the final lipid
organized structure
A phospholipid
Lipid Organization
Phopholipid Hierarchal Structures
Liposomes
24. Liposomes
• Liposomes are
– not limited by size or number of genes
– safe
– easy to produce
– short-term expression
26. • Diverse manners of ‘lysing’ the liposome
• Temperature sensitive
• Target sensitive
• pH sensitive
• Electric field sensitive
Liposomes
27. Limitations of Gene Therapy
• Gene delivery
– Limited tropism of viral vectors
– Dependence on cell cycle by some viral vectors
(i.e. mitosis required)
• Duration of gene activity
– Non-integrating delivery will be transient (transient
expression)
– Integrated delivery will be stable
• Patient safety
– Immune hyperresponsiveness (hypersensitivity
reactions directed against viral vector components
or against transgenes expressed in treated cells)
– Integration is not controlled oncogenes may be
involved at insertion point cancer?
28. • Gene control/regulation
– Most viral vectors are unable to accommodate full
length human genes containing all of their original
regulatory sequences
– Human cDNA often used much regulatory
information is lost (e.g. enhancers inside introns)
– Often promoters are substituted therefore gene
expression pattern may be very different
– Random integration can adversely affect
expression (insertion near highly methylated
heterogeneous DNA may silence gene
expression)
Limitations of Gene Therapy
29. • Expense
– Costly because of cell
culturing needs involved
in ex vivo techniques
– Virus cultures for in vivo
delivery
– Usually the number of
patients enrolled in any
given trial is <20
– More than 5000 patients
have been treated in last
~12 years worldwide
Limitations of Gene Therapy
3Other
196Cancer
21HIV
18Genetic disease
# Trials (total =
338)
Diagnosis
Gene Therapy Trials in U.S.
(Information from US NIH, Office
of
Recombinant DNA Activities –
1999)
31. Diseases for applying gene therapy
Disease Defect Target cell
Severe combined Bone marrow cells or
immunodeficiency T-lymphocytes
Hemophilia Liver, muscle
Cystic fibrosis Lung Cells
Cancer Many cell types
Neurological diseases Parkinson’s/ Alzheimers Nerve Cells
Infectious diseases AIDS, hepatitis B White Blood Cells
32. Gene therapy could be
very different for different diseases
• Gene transplantation
(to patient with gene deletion)
• Gene correction
(To revert specific mutation in the gene of interest)
• Gene augmentation
(to enhance expression of gene of interest)
33. Cystic fibrosis
most common lethal genetic disorder in Caucasian populations
(1 in 2000 live births.) . Among African and Asian is really rare
a defect in the CFTR gene
Lungs create
thick mucus secretion
(prone to infections,
constant cough,
leading cause of death)
34. Lungs in cystic fibrosis
Normal lung CF lungs
dilated crypts
filled with mucus and
bacteria.
Normal alveolar appearance
CF lungs filled with mucus
lung did not collapse
when it was removed
postmortem
35. Example: Severe Combined
Immunodeficiency Disease (SCID)
• Before GT, patients received a bone marrow
transplant
– David, the “Boy in the Bubble”, received BM from
his sister unfortunately he died from a a form
of blood cancer
36. What is Severe Combined
Immunodoficiency (SCID)?
> 8 new ear infections per year
> 2 serious sinus infections per year
> 2 month on antibiotics with little effect
> 2 pneumonias per year
-- failure to gain weight and grow
-- recurrent deep skin and organ abscesses
37. • SCID is caused by an Adenosine Deaminase
Deficiency (ADA)
– Gene is located on chromosome #22 (32 Kbp, 12
exons)
– Deficiency results in failure to develop functional T and
B lymphocytes
– ADA is involved in purine degradation
– Accumulation of nucleotide metabolites = TOXIC to
developing T lymphocytes
– B cells don’t mature because they require T cell help
– Patients cannot withstand infection die if untreated
38. • September 14, 1990 @ NIH, French Anderson and
R. Michael Blaese perform the first GT Trial
– Ashanti (4 year old girl)
• Her lymphocytes were gene-altered (~109
) ex
vivo used as a vehicle for gene introduction
using a retrovirus vector to carry ADA gene
(billions of retroviruses used)
– Cynthia (9 year old girl) treated in same year
• Problem: WBC are short-lived, therefore treatment
must be repeated regularly
39. Parkinson's Disease Cont.
• The gene transfer procedure utilized the
AAV (adeno-associated virus) vector, a
virus that has been used safely in a variety
of clinical gene therapy trials, and the
vehicle that will be used in all of the
company's first generation products,
including epilepsy and Huntington's
disease. In its Parkinson's disease trial,
Neurologix used its gene transfer
technology.
40.
41. Ornithine transcarbamylase (OTC)
deficiency
• September 17, 1999
– Ornithine transcarbamylase (OTC) deficiency
• Urea cycle disorder (1/10,000 births)
• Encoded on X chromosome
– Females usually carriers, sons have
disease
– Urea cycle = series of 5 liver enzymes that rid the
body of ammonia (toxic breakdown product of
protein)
• If enzymes are missing or deficient, ammonia
accumulates in the blood and travels to the
brain (coma, brain damage or death)
42. • Severe OTC deficiency
– Newborns coma within 72 hours
• Most suffer severe brain damage
• ½ die in first month
• ½ of survivors die by age 5
– Early treatment
• Low-protein formula called “keto-acid”
– Modern day treatment
• Sodium benzoate and another sodium
derivative
• Bind ammonia helps eliminate it from the
body
Ornithine transcarbamylase (OTC)
deficiency
43. • Case study: Jesse Gelsinger
– GT began Sept. 13, 1999, Coma on Sept. 14, Brain
dead and life support terminated on Sept. 17, 1999
– Cause of death: Respiratory Disease Syndrome
– Adenovirus (a weakened cold virus) was the vector of
choice (DNA genome and an icosahedral capsid)
– Chain reaction occurred that previous testing had not
predicted following introduction of “maximum tolerated
dose”
• Jaundice, kidney failure, lung failure and brain
death
• Adenovirus triggered an overwhelming inflammatory
reaction massive production of monokine IL-6
multiple organ failure
Ornithine transcarbamylase (OTC)
deficiency
44. • AIDS
– HIV patients T lymphocytes treated ex vivo
with rev and env defective mutant strains of HIV
– Large numbers of cells obtained
• Injected back into patient
• Stimulated good CD8+
cytotoxic T cell
responses (Tcyt)
45. • Familial Hypercholesterolemia
– Defective cholesterol receptors on liver cells
• Fail to filter cholesterol from blood properly
• Cholesterol levels are elevated, increasing risk
of heart attacks and strokes
– 1993 First attempt
• Retroviral vector used to infect 3.2 x 109
liver
cells (~15% of patients liver) ex vivo
– Infused back into patient
– Improvement seen
– Has been used in many trials since then
46. • Lesch-Nyhan Disease – Candidate
– Early days confined to animal models and in vitro
tests
– Defect in producing HGPRT enzyme (hypoxanthine-
guanine phosphoribosyl transferase)
• Defective metabolism of hypoxanthine and guanine
Uric acid accumulates
– Gout, Kidney disease, cerebral palsy, mental
retardation, head banging, profanity, spitting,
mutilation of fingers
47. • Gaucher’s disease
– Glucocerebrosidase gene defect
– RAC approved clinical tests – 1993
– Affects CNS, enlarged spleen and liver, long
bone erosion and discoloration of skin
48. • Liposomes coated in polymer PEG – can cross the
blood-brain barrier (viral vectors are too big) (January
2003)
• Case Western Uni. & Copernicus Therapeutics able
to create tiny liposomes 25nm across to carry
therapeutic DNA through pores in nuclear membrane
• New gene approach repairs errors in mRNA
• Thalassaemia
• Cystic fibrosis
• Some cancers
Recent Developments in Gene Therapy
49. • 2003 – temporary hold on all gene therapy trials
including retroviral vectors in blood stem cells
• Too early to tell
• Desperately need improved DELIVERY …could
liposomes be the answer?
Future?
50. • 2003 – temporary hold on all gene therapy trials
including retroviral vectors in blood stem cells
• Too early to tell
• Desperately need improved DELIVERY …could
liposomes be the answer?
Future?