Gene therapy lecture_spring_2007
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Gene therapy aims to manage and correct human diseases like cancer, AIDS, and inherited disorders. While promising advances have been made in DNA technology, the efficacy of gene therapy protocols has yet to be definitively proven due to insufficient understanding of vector-host interactions. Gene therapy works by delivering exogenous DNA using a viral vector to enable protein expression in target cells. However, barriers like the immune system and lysosomes can prevent successful gene transfer. While many clinical applications exist, such as for inherited monogenic disorders and infectious diseases, safety issues remain a challenge as evidenced by the death of the first patient to die from a gene therapy treatment.
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Gene therapy
Gene therapy is an experimental technique that involves inserting a normal gene to replace a defective gene responsible for disease. It can replace a mutated disease-causing gene with a healthy copy or inactivate a mutated gene functioning improperly. Common diseases targeted include severe combined immunodeficiency, hemophilia, cystic fibrosis, hemoglobinopathies like thalassemia and sickle cell anemia, Parkinson's disease, and cancer. The first approved gene therapy case occurred in 1990 treating a girl with immune deficiency. Since then, gene therapy has been used to treat metastatic melanoma. Gene therapy can affect the germline permanently or somatic cells temporarily.
Applications of gene therapy
Applications of gene therapy and potential disease targeted by gene therapy ( Basic Information about them)
GENE THERAPY: PRINCIPLES, PROBLEMS AND PROSPECTS
This document summarizes a seminar presentation on gene therapy. It discusses the basic principles of genes and gene therapy, including how genes encode proteins and how mutations can cause disease. It describes the two main types of gene therapy - somatic and germline. It also outlines some viral vectors used, methods of gene transfer like ex vivo and in vivo, diseases that may be treatable with gene therapy, prospects of the approach, and concludes that further development is needed but gene therapy has potential to benefit humanity.
GENE THERAPHY
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
Gene therapy holds promise for treating diseases like cancer, HIV, and genetic disorders. It works by inserting a normal gene to replace an abnormal one causing disease. Viruses are often used as vectors to deliver the new gene into cells. There are two main types: germline alters genes in reproductive cells and is unethical, while somatic only alters genes in other body cells. Gene therapy has had some success but also deaths, and faces ethical issues regarding cost, safety, and enhancement versus treatment of disease. It remains experimental but could help many if developed responsibly.
Gene therapy in clinical applications
Gene therapy.
Adenovirus
Retrovirus
In vivo and Ex vivo methods
Molecular techniques
Alternative vectors
Biolistic missile
Homologous recombination
Gene therapy
this presentation about. ex- vivo and in-vivo gene therapy with ex. of cystic fibrosis and SCID disease.
Gene therapy
GENE THERAPY
S.RAM PRAKASH
IIIrdBSC BIOTECHNOLOGY, AYYA NADAR JANAKI AMMA COLLEGE, SIVAKASI, TAMIL NADU.
Recommended
Gene therapy
Gene therapy is an experimental technique that involves inserting a normal gene to replace a defective gene responsible for disease. It can replace a mutated disease-causing gene with a healthy copy or inactivate a mutated gene functioning improperly. Common diseases targeted include severe combined immunodeficiency, hemophilia, cystic fibrosis, hemoglobinopathies like thalassemia and sickle cell anemia, Parkinson's disease, and cancer. The first approved gene therapy case occurred in 1990 treating a girl with immune deficiency. Since then, gene therapy has been used to treat metastatic melanoma. Gene therapy can affect the germline permanently or somatic cells temporarily.
Applications of gene therapy
Applications of gene therapy and potential disease targeted by gene therapy ( Basic Information about them)
GENE THERAPY: PRINCIPLES, PROBLEMS AND PROSPECTS
This document summarizes a seminar presentation on gene therapy. It discusses the basic principles of genes and gene therapy, including how genes encode proteins and how mutations can cause disease. It describes the two main types of gene therapy - somatic and germline. It also outlines some viral vectors used, methods of gene transfer like ex vivo and in vivo, diseases that may be treatable with gene therapy, prospects of the approach, and concludes that further development is needed but gene therapy has potential to benefit humanity.
GENE THERAPHY
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
Gene therapy holds promise for treating diseases like cancer, HIV, and genetic disorders. It works by inserting a normal gene to replace an abnormal one causing disease. Viruses are often used as vectors to deliver the new gene into cells. There are two main types: germline alters genes in reproductive cells and is unethical, while somatic only alters genes in other body cells. Gene therapy has had some success but also deaths, and faces ethical issues regarding cost, safety, and enhancement versus treatment of disease. It remains experimental but could help many if developed responsibly.
Gene therapy in clinical applications
Gene therapy.
Adenovirus
Retrovirus
In vivo and Ex vivo methods
Molecular techniques
Alternative vectors
Biolistic missile
Homologous recombination
Gene therapy
this presentation about. ex- vivo and in-vivo gene therapy with ex. of cystic fibrosis and SCID disease.
Gene therapy
GENE THERAPY
S.RAM PRAKASH
IIIrdBSC BIOTECHNOLOGY, AYYA NADAR JANAKI AMMA COLLEGE, SIVAKASI, TAMIL NADU.
Gene therapy
This document provides an overview of gene therapy. It discusses the definition of gene therapy as inserting genes into cells to treat disease. The history of gene therapy is described, including early research in the 1970s and the first human clinical trial in 1990 to treat ADA deficiency. The document outlines the main types of gene therapy as somatic, germline, and preventative. It also discusses gene delivery methods using viral and non-viral vectors and the advantages and disadvantages of gene therapy.
GENE THERAPY
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,
Strategies of gene therapy
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.
Success of gene therapy
Gene therapy has shown success in treating various diseases. The global gene therapy market is growing rapidly, with many companies raising funds. Gene therapy is significantly helping treat diseases like cancer, cardiovascular disease, and genetic disorders. While gene therapy has made progress, there are still challenges to overcome like short duration of effect, immune responses, safety of viral vectors, and ethical issues. Overall, gene therapy is a promising area that is helping treat previously untreatable conditions.
Gene therapy
These slide include gene therapy defines with their types like Germ line gene therapy,Somatic gene therapy.
with Need of Gene therapy
strategies of gene therapy
Methods of Gene transfer & with
GENE THERAPY FOR INHERITED DISORDERS
About The Gene Therapy
Gene therapy is an experimental technique that uses genes to treat or prevent disease. The slides explain what is gene tharapy? Types of gene therapy. http://www.wesrch.com/
Gene delivery and gene therapy . Gene delivery , Gene therapy .
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 for Biotechnologist
This document discusses gene therapy, including its history, mechanisms, applications, challenges, and recent developments. It provides an overview of the first human gene therapy trials in 1990 for severe combined immunodeficiency, as well as both successful and unsuccessful early gene therapy cases. Recent progress includes using gene therapy to potentially treat Parkinson's disease, cancer, blood disorders, and inherited blindness. Overall, the document outlines the key concepts and timeline of gene therapy from its beginnings to current research.
Gene therapy ppt for students and staff
Surviving animals produced by nuclear transfer are healthy.
There, is a substantial loss of individual before and after birth some of the cloned animals display abnormalities.
Abnormlities such as increased birth weight.
Dna methylation and histone modification of the original donor cell is inappropriate maintained in the cells of the recipient animals.
INVIVO:
The direct delivery of the therapeutic gene into the target cells of a particular tissue.
Genes are changed in cells when the cells are still in the body.
These include liver, muscle, skin, spleen, lung, brain and blood cells.
Carried out by viral and non viral vector system
EXVIVO:
Cells are modified outside the body and then transplanted back in again.
SCID is a inherited immune disorder associated with T-lymphocyte and B-lymphocytes dysfunction.
Gene encodes for adenosine deaminase.
The patients of SCID(lacking ADA) suffer from infectious diseases and die at an young age.
Gene therapy
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.
Gene therapy
This document discusses gene therapy and its various aspects. It defines gene therapy as a method of treatment using genes or DNA instead of drugs to treat diseases caused by defective genes. The DNA is delivered into cells using vectors like viruses. The document discusses two main types of gene therapy - somatic cell therapy which affects only the treated individual, and germ line therapy which affects future generations. It also discusses various gene therapy strategies, vectors, methods of gene delivery and challenges in gene therapy.
Gene therapy
Gene therapy involves using DNA as a pharmaceutical to treat disease. It works by removing cells from a patient, exposing them to viruses carrying desired genes, and regrowing the modified cells to be injected back into the patient. While it has successfully treated some diseases, gene therapy also carries risks like causing new conditions if viruses replicate improperly. The document outlines the history, techniques, applications, and ethical considerations of gene therapy.
The Use of Gene Therapy to Treat Type 1 Diabetes Mellitus
This research proposal aims to use gene therapy to prevent the rejection of islet cell transplants in type 1 diabetes patients. The researcher hypothesizes that by adding an immunosuppressive gene to the transplanted islet cells using an adeno-associated viral vector, the immune system will not attack the cells and endogenous insulin production can occur without other immunosuppressive drugs. The proposal involves testing this method on AKITA mice with diabetes by transplanting islet cells containing the CD47 gene or no added gene, and monitoring blood sugar levels to compare effectiveness of the transplanted cells at regulating blood sugar. The researcher expects the mice receiving cells with the CD47 gene will show greater insulin production and better blood sugar control.
Gene therapy - Dr.Padmesh
Gene therapy involves introducing functional genes into patients' cells to treat diseases caused by defective genes. There are two main approaches - ex vivo, where cells are modified outside the body then reintroduced, and in vivo, where genes are directly inserted into tissues. Common gene delivery methods include viral vectors like retroviruses and adenoviruses, as well as non-viral methods like liposomes. While early gene therapy trials showed promise for diseases like severe combined immunodeficiency, risks remain such as adverse immune reactions and insertional mutagenesis.
Gene theapy
This document provides an overview of gene therapy and its applications in cancer, HIV/AIDS, and hereditary diseases. It discusses how gene therapy works by using vectors to deliver therapeutic genes into target cells to repair faulty genes. For cancer, strategies discussed include enhancing immune response, inserting suicide genes, and blocking oncogenes. For HIV/AIDS, intracellular immunization, ribozymes, transdominant mutants, and Trojan horses are described as strategies. For hereditary diseases, successes in clinical trials for SCID and hemophilia are summarized, with future trials planned for other genetic disorders.
Gene Therapy MANIK
Gene Therapy: Central concept of gene therapy, basic molecular mechanism of gene transfer, prerequisite of human gene therapy, biological basis of gene therapy strategies, vehicles for gene transfer, Antisence oligonucleotides and RNAi, clinical gene therapy studies, gene therapy for hereditary disease, gene therapy for cancer, gene therapy for HIV.
Gene therapy
"Gene Therapy" is a technique used to treat a disease or disorder by inducing the therapeutic gene into the vector, and the vector into the affected organism. So that, the therapeutic proteins expressed will vanish the affected proteins.
Gene therapy
Basic principles of genetics and the basic understanding of gene therapy and its applications in therapeutics.
Gene therapy
Gene therapy involves inserting functioning genes into patients' cells to treat genetic diseases. It has been studied as a potential treatment for cancers, blood diseases, immune and neurological disorders. There are two main types - somatic cell gene therapy targets specific cells/tissues, while germline gene therapy affects heritable genetic material. Recent progress includes the first approved gene therapy in 1990 and advancements in viral vectors. Major milestones in the 2000s-2010s included treatments for leukemia, ADA-SCID, and retinal diseases. Future prospects are promising with CRISPR/Cas9 offering more precise gene editing and many ongoing clinical trials, though challenges remain around costs, technical expertise, and ethical concerns.
Gene therapy
Gene therapy involves introducing genetic material into host cells to treat diseases caused by genetic mutations. It has been used to treat several conditions like ADA-SCID, cystic fibrosis, and inherited retinal diseases. Various gene delivery systems exist including viral vectors like adenovirus, AAV, and retroviruses. Strategies for gene therapy include gene augmentation, inhibition, targeting, assisted killing, and prodrug therapy. While promising, gene therapy still faces challenges like improving delivery methods and reducing immune responses.
Transgenic cetp dahl salt sensitive (tg53) rat model
Transgenic cetp dahl salt sensitive (tg53) rat modelSociety for Heart Attack Prevention and Eradication
The document discusses the potential of the Tg53 rat model for modeling human coronary artery disease and vulnerable plaques. The model shows promise because it develops plaques specifically in the coronary arteries, like in humans, and the plaques progress through stages and demonstrate features resembling vulnerable human plaques. The model also has advantages as an experimental system, including reproducibility, defined genetic background, and ability to manipulate the disease course. The document argues the model has value for investigating mechanisms of plaque development and testing novel interventions, and could serve as a promising pre-clinical platform when used together with other models.Fish glow green after genetic engineering
Genetically engineered zebrafish that glow green under fluorescence help scientists track how pollutants affect the body. When exposed to chemicals like bisphenol A, different organs of the fish like the heart and eyes glow, illuminating what tissues are impacted. This helps researchers better understand how these endocrine disrupting chemicals mimic hormones and cause issues like changing fish gender and links to cancers in people. The fluorescent technology is currently limited to fish under 6 days old, but future work aims to breed fish without skin pigment to observe reactions in adult fish as well.
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Gene therapy
This document provides an overview of gene therapy. It discusses the definition of gene therapy as inserting genes into cells to treat disease. The history of gene therapy is described, including early research in the 1970s and the first human clinical trial in 1990 to treat ADA deficiency. The document outlines the main types of gene therapy as somatic, germline, and preventative. It also discusses gene delivery methods using viral and non-viral vectors and the advantages and disadvantages of gene therapy.
GENE THERAPY
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,
Strategies of gene therapy
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.
Success of gene therapy
Gene therapy has shown success in treating various diseases. The global gene therapy market is growing rapidly, with many companies raising funds. Gene therapy is significantly helping treat diseases like cancer, cardiovascular disease, and genetic disorders. While gene therapy has made progress, there are still challenges to overcome like short duration of effect, immune responses, safety of viral vectors, and ethical issues. Overall, gene therapy is a promising area that is helping treat previously untreatable conditions.
Gene therapy
These slide include gene therapy defines with their types like Germ line gene therapy,Somatic gene therapy.
with Need of Gene therapy
strategies of gene therapy
Methods of Gene transfer & with
GENE THERAPY FOR INHERITED DISORDERS
About The Gene Therapy
Gene therapy is an experimental technique that uses genes to treat or prevent disease. The slides explain what is gene tharapy? Types of gene therapy. http://www.wesrch.com/
Gene delivery and gene therapy . Gene delivery , Gene therapy .
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 for Biotechnologist
This document discusses gene therapy, including its history, mechanisms, applications, challenges, and recent developments. It provides an overview of the first human gene therapy trials in 1990 for severe combined immunodeficiency, as well as both successful and unsuccessful early gene therapy cases. Recent progress includes using gene therapy to potentially treat Parkinson's disease, cancer, blood disorders, and inherited blindness. Overall, the document outlines the key concepts and timeline of gene therapy from its beginnings to current research.
Gene therapy ppt for students and staff
Surviving animals produced by nuclear transfer are healthy.
There, is a substantial loss of individual before and after birth some of the cloned animals display abnormalities.
Abnormlities such as increased birth weight.
Dna methylation and histone modification of the original donor cell is inappropriate maintained in the cells of the recipient animals.
INVIVO:
The direct delivery of the therapeutic gene into the target cells of a particular tissue.
Genes are changed in cells when the cells are still in the body.
These include liver, muscle, skin, spleen, lung, brain and blood cells.
Carried out by viral and non viral vector system
EXVIVO:
Cells are modified outside the body and then transplanted back in again.
SCID is a inherited immune disorder associated with T-lymphocyte and B-lymphocytes dysfunction.
Gene encodes for adenosine deaminase.
The patients of SCID(lacking ADA) suffer from infectious diseases and die at an young age.
Gene therapy
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.
Gene therapy
This document discusses gene therapy and its various aspects. It defines gene therapy as a method of treatment using genes or DNA instead of drugs to treat diseases caused by defective genes. The DNA is delivered into cells using vectors like viruses. The document discusses two main types of gene therapy - somatic cell therapy which affects only the treated individual, and germ line therapy which affects future generations. It also discusses various gene therapy strategies, vectors, methods of gene delivery and challenges in gene therapy.
Gene therapy
Gene therapy involves using DNA as a pharmaceutical to treat disease. It works by removing cells from a patient, exposing them to viruses carrying desired genes, and regrowing the modified cells to be injected back into the patient. While it has successfully treated some diseases, gene therapy also carries risks like causing new conditions if viruses replicate improperly. The document outlines the history, techniques, applications, and ethical considerations of gene therapy.
The Use of Gene Therapy to Treat Type 1 Diabetes Mellitus
This research proposal aims to use gene therapy to prevent the rejection of islet cell transplants in type 1 diabetes patients. The researcher hypothesizes that by adding an immunosuppressive gene to the transplanted islet cells using an adeno-associated viral vector, the immune system will not attack the cells and endogenous insulin production can occur without other immunosuppressive drugs. The proposal involves testing this method on AKITA mice with diabetes by transplanting islet cells containing the CD47 gene or no added gene, and monitoring blood sugar levels to compare effectiveness of the transplanted cells at regulating blood sugar. The researcher expects the mice receiving cells with the CD47 gene will show greater insulin production and better blood sugar control.
Gene therapy - Dr.Padmesh
Gene therapy involves introducing functional genes into patients' cells to treat diseases caused by defective genes. There are two main approaches - ex vivo, where cells are modified outside the body then reintroduced, and in vivo, where genes are directly inserted into tissues. Common gene delivery methods include viral vectors like retroviruses and adenoviruses, as well as non-viral methods like liposomes. While early gene therapy trials showed promise for diseases like severe combined immunodeficiency, risks remain such as adverse immune reactions and insertional mutagenesis.
Gene theapy
This document provides an overview of gene therapy and its applications in cancer, HIV/AIDS, and hereditary diseases. It discusses how gene therapy works by using vectors to deliver therapeutic genes into target cells to repair faulty genes. For cancer, strategies discussed include enhancing immune response, inserting suicide genes, and blocking oncogenes. For HIV/AIDS, intracellular immunization, ribozymes, transdominant mutants, and Trojan horses are described as strategies. For hereditary diseases, successes in clinical trials for SCID and hemophilia are summarized, with future trials planned for other genetic disorders.
Gene Therapy MANIK
Gene Therapy: Central concept of gene therapy, basic molecular mechanism of gene transfer, prerequisite of human gene therapy, biological basis of gene therapy strategies, vehicles for gene transfer, Antisence oligonucleotides and RNAi, clinical gene therapy studies, gene therapy for hereditary disease, gene therapy for cancer, gene therapy for HIV.
Gene therapy
"Gene Therapy" is a technique used to treat a disease or disorder by inducing the therapeutic gene into the vector, and the vector into the affected organism. So that, the therapeutic proteins expressed will vanish the affected proteins.
Gene therapy
Basic principles of genetics and the basic understanding of gene therapy and its applications in therapeutics.
Gene therapy
Gene therapy involves inserting functioning genes into patients' cells to treat genetic diseases. It has been studied as a potential treatment for cancers, blood diseases, immune and neurological disorders. There are two main types - somatic cell gene therapy targets specific cells/tissues, while germline gene therapy affects heritable genetic material. Recent progress includes the first approved gene therapy in 1990 and advancements in viral vectors. Major milestones in the 2000s-2010s included treatments for leukemia, ADA-SCID, and retinal diseases. Future prospects are promising with CRISPR/Cas9 offering more precise gene editing and many ongoing clinical trials, though challenges remain around costs, technical expertise, and ethical concerns.
Gene therapy
Gene therapy involves introducing genetic material into host cells to treat diseases caused by genetic mutations. It has been used to treat several conditions like ADA-SCID, cystic fibrosis, and inherited retinal diseases. Various gene delivery systems exist including viral vectors like adenovirus, AAV, and retroviruses. Strategies for gene therapy include gene augmentation, inhibition, targeting, assisted killing, and prodrug therapy. While promising, gene therapy still faces challenges like improving delivery methods and reducing immune responses.
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The Use of Gene Therapy to Treat Type 1 Diabetes Mellitus
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Gene therapy
Mrs. B.Gomathi, Associate Professor and Head, Dept of OBG Nursing, SUM Nursing College, Shiksha 'O' Anusandhan Deemed to be University, Bhubaneswar.
Gene therapy
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 1
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.
Genetherapy : An Overview
Gene therapy involves the insertion of a functioning gene into cells to correct a cellular dysfunction
KEY WORDS : GENETICS, MUTATION , GENETIC ENGINEERING.
Human genome project 2007
The human genome project mapped the entire human genetic code and identified approximately 30,000 human genes, providing insights into the genetic basis of diseases and opportunities for new diagnostic tests and treatments. Gene therapy aims to treat diseases by modifying genes, either in vivo by introducing normal genes into patients' cells to replace mutated genes or ex vivo by extracting cells, modifying them genetically, and returning them to patients. While promising, gene therapy faces challenges in safety, delivery methods, and treating complex multi-gene disorders.
Gene therapy.pptxjjjjnnnsnskskslsllssnnznzj
Gene therapy involves inserting genes into an individual's cells and tissues to treat disease. It can replace mutated genes, inactivate genes, introduce new genes, or cause cancer cells to kill themselves. Viral and non-viral vectors are used to deliver genes. Gene therapy has been applied to treat genetic disorders, cancer, heart disease, and more. Recent advances include using gene therapy to regenerate heart muscle cells, treat Sanfilippo syndrome and brain cancers, and combining cellular and gene therapies for breast cancer. RNA and DNA can be estimated using reactions that form colored complexes measured spectrophotometrically.
Gene rx [autosaved]
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.
Gene therapy final
The document provides an overview of gene therapy, including:
1) Gene therapy involves using genes to treat genetic diseases by techniques like inserting normal genes to replace abnormal ones.
2) Gene therapy classifications include gene replacement, gene deactivation, transgenesis, and gene enhancement therapies.
3) Viral vectors like retroviruses and adenoviruses are commonly used for gene delivery but pose risks like immune reactions.
Gene Therapy
Gene therapy involves inserting functional genes into patients' cells to treat genetic diseases. It was first proposed in the 1960s and attempted in the 1970s. The first approved human gene therapy trial took place in 1990, treating a girl with severe combined immunodeficiency. Since then, gene therapy has been used experimentally to treat various diseases including cancer, cystic fibrosis, and HIV. There are two main approaches - ex vivo, where cells are removed and modified before being returned to the body, and in vivo, where genes are directly inserted into target tissues. Viruses are often used as vectors to deliver therapeutic genes, with retroviruses commonly used for ex vivo therapy and adenoviruses for in vivo.
Gene Therapy Manik
Gene Therapy: Central concept of gene therapy, basic molecular mechanism of gene transfer, prerequisite of human gene therapy, biological basis of gene therapy strategies, vehicles for gene transfer, Antisence oligonucleotides and RNAi, clinical gene therapy studies, gene therapy for hereditary disease, gene therapy for cancer, gene therapy for HIV.
Gene therapy.
This document outlines a seminar topic on somatic cell gene therapy. It discusses the different types of gene therapy, including somatic cell gene therapy and germ line gene therapy. Somatic cell gene therapy involves transferring genes into any cell other than germ cells to treat an individual patient only. The document then describes the various types of somatic cell gene therapy in more detail, such as ex-vivo gene therapy, in-vivo gene therapy using viruses, and antisense gene therapy. It also reviews some advantages and disadvantages of gene therapy.
Gene Therapy Resala
The document discusses gene therapy as a promising approach for treating various diseases. It provides a brief history of gene therapy and describes some of the early clinical trials. It then explains some of the key concepts in medical genetics like DNA, genes, and enzymes. Different methods for gene delivery are also summarized, including viral vectors, physical methods, and chemical methods. Applications of gene therapy for cancer, neurological disorders, and other diseases are briefly mentioned.
Gene Therapyrr
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
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.
Human gene therapy
Gene Therapy, Somatic cell gene therapy, germ line gene therapy, classical gene therapy, non-classical gene therapy, targets of gene therapy, barriers of gene therapy, ex vivo gene therapy, in vivo gene therapy, vectors for gene delivery, antisense therapy
Gene medicine by kk sahu sir
INTRODUCTION
DNA VACCINES
GENE THERAPY
TIME LINE OF DEVELOPING GENE THERAPY
GENE THERAPY STRATEGIES
TECHNOLOGY OF CLASSICAL GENE THERAPY
PRINCIPLES OF GENE TRANSFER
VECTORS
VIRAL VECTORS
NON-VIRAL VECTORS
APPLICATIONS OF GENE THERAPY
ETHICAL IMPLICATIONS
THE FUTURE
CONCLUSION
REFERENCES
Gene therapy advanced treatments for a new era aranca special report
Aranca's Report on Gene Therapy - a promising tool for Cancer, Parkinson's, HIV, severe combined immuno-deficiencies, hemophilia etc. In this report, you will discover the challenges associated with Gene Therapy as well as its expected future.
GENE THERAPY
This document provides an overview of gene therapy. It defines gene therapy as an experimental technique for correcting defective genes responsible for disease. It describes the main approaches like somatic cell gene therapy and germline gene therapy. It also discusses viral and non-viral vectors, delivery methods like in vivo and ex vivo, advantages like curing genetic diseases, and challenges like short-term effects and safety issues. Recent developments show promise for treating diseases like blindness and Parkinson's.
1. gene therapy
Gene therapy involves inserting normal genes into a person's cells to treat a disease. There are several strategies for gene therapy, including ex vivo and in vivo methods. Viral vectors are commonly used to deliver therapeutic genes to target cells. However, gene therapy has faced challenges such as short-lived effects, immune responses, and safety issues with viral vectors. Continued research is working to overcome these issues and further develop promising applications of gene therapy.
Gene therapy
Gene therapy involves inserting a normal gene into a patient's cells to replace a defective gene that causes disease. There are two main types of gene therapy: somatic cell therapy, which treats the individual patient, and germline therapy, which could affect future generations. The two most common methods of delivery are ex vivo therapy, where cells are removed and modified before being returned, and in vivo therapy through direct injection. Retroviruses and adenoviruses are the most widely used vectors for transporting genes, but each has advantages and disadvantages for successful gene expression and integration. While promising for treating many diseases, gene therapy still faces challenges from issues like gene silencing, immunogenicity, and potential safety risks that require more research.
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Gene therapy advanced treatments for a new era aranca special report
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Gene therapy lecture_spring_2007
- 1. APPLICATIONS FOR GENE THERAPY Felix Kriedemann Gonzalo Briones 11ºA 1
- 2. Gene Therapy Objectives: 1. Recognize significance and uses of gene therapy. 2. Define background theory and methodologies. 3. Address potential clinical applications, limitations, and future directions. 2
- 3. Objectives of gene therapy: 1. Management and correction of human diseases a. Inherited and acquired disorders b. Cancer c. AIDS/HIV Good news: Promising advances during the last two decades in making DNA technology. Bad news: Efficacy in any gene therapy protocol not definitive. 1. Inadequate understanding of biological interactions of vector and host. 3
- 4. (27) (86) 4
- 5. 5
- 6. Some global terms/definitions: 1. Genes and nucleic acids 2. Vector, viral delivery systems 3. Gene transfer 4. Reporter gene 5. Transfer efficiency 6. Germ vs. somatic cells 7. In/ex vivo, in situ, in vitro. 6
- 7. Genetic diseases: Type 1: Single locus (gene) is defective and responsible for the disease, 100% heritable. examples: Sickle cell anemia, Hypercholesterolemia Cystic fibrosis Type 2: Polygenic traits, less than 100% heritable, may be dependent on environmental factors and lifestyle. examples: Heart disease Cancer Diabetes Alcoholism Schizophrenia Criminal behavior? etc….? 7
- 8. How it works ? Exogenous DNA + vector (viral) Protein expression Cytosol Endosome Transcription Lysosome (nucleases) Barriers that prevent transfer of exogenous DNA 8
- 9. Categories of clinical gene transfer protocols. 1. Inherited/monogenic disorders: ADA deficiency Alpha-1 antitrypsin Chronic granulomatous disease Cystic fibrosis Familial hypercholesterolemia Fanconi Anemia Gaucher Disease Hunter syndrome Parkinsons 2. Infectious Diseases: HIV 9
- 10. 4. Cancer (by approach): Antisense Chemoprotection Immunotherapy: ex vivo / in vivo Thymidylate kinase Tumor suppressor genes 3. Acquired disorders: peripheral artery disease Rheumatoid arthritis 10
- 11. 11
- 12. ‘Cancer regression in patients after transfer of genetically engineered lymphocytes.’ Liver Lymph node 12
- 13. Case study: Jesse Gelsinger *First documented patient to die from gene therapy treatment. Disease: liver enzyme deficiency (ornithine transcarbamylase, OTC) – controls ammonia metabolism Vector used to deliver OTC – modified adenovirus Objective: deliver vector to liver cells and express OTC. Problem: Very low transfer efficiency (1%), difficult to get enough functioning OTC expressed to do any good. Solution: Infect with higher dose of viral particles. (38 trillion) 13
- 14. Outcome: -Vector not only delivered gene to liver but to other tissue. - Systemic inflammatory response. -Patient acquired fever, coma and finally death. Why? -Animal studies suggested that the dose was correct . -Adenoviral vectors known to induce inflammatory response. -Patient already compromised: Patient had higher than allowed ammonia levels. 14
- 15. Bibliography And, A. (1994). Gene Therapy: Some History, Applications, Problems, and Prospects. [online] Retrieved from: http://tpx.sagepub.com/content/36/1/97.full [Accessed: 2 Apr 2013]. Herkules.oulu.fi (1997). Applications of somatic gene therapy. [online] Retrieved from: http://herkules.oulu.fi/isbn9514255429/html/x298.html [Accessed: 2 Apr 2013]. Intechopen.com (2012). What is Gene Therapy | Applications | InTechOpen. [online] Retrieved from: http://www.intechopen.com/books/gene-therapy- applications [Accessed: 2 Apr 2013]. Iptv.org (2004). Gene Therapy » Medical Uses of GE » Uses » Explore More: Genetic Engineering. [online] Retrieved from: http://www.iptv.org/exploremore/ge/uses/use2_gene.cfm [Accessed: 2 Apr 2013]. MedicineNet (2013). Gene Therapy - The Future Is Here!. [online] Retrieved from: http://www.medicinenet.com/script/main/art.asp?articlekey=12662 [Accessed: 2 Apr 2013]. YouTube (n.d.). Vector design for vaccine and gene therapy applications. [online] Retrieved from: http://www.youtube.com/watch?v=zKLpRufcy0k [Accessed: 2 Apr 2013]. 15
Editor's Notes
- SCID = severe combined immunodeficiency.