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 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.
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.
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.
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.
Gene therapy is a strategy to treat disease by correcting defective genes or modifying gene expression. The first approved gene therapy experiment occurred in 1990 and treated a patient with ADA-SCID by inserting genes into their cells. There are two main approaches to gene therapy - in vivo and ex vivo. In vivo therapy involves direct injection of vectors into the body, while ex vivo therapy genetically modifies cells outside the body before reintroducing them. Viral vectors like adenoviruses are commonly used to deliver therapeutic genes, but carry risks like immune responses.
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 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.
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.
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.
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.
Gene therapy is a strategy to treat disease by correcting defective genes or modifying gene expression. The first approved gene therapy experiment occurred in 1990 and treated a patient with ADA-SCID by inserting genes into their cells. There are two main approaches to gene therapy - in vivo and ex vivo. In vivo therapy involves direct injection of vectors into the body, while ex vivo therapy genetically modifies cells outside the body before reintroducing them. Viral vectors like adenoviruses are commonly used to deliver therapeutic genes, but carry risks like immune responses.
Gene therapy involves introducing functional genes into patients to treat diseases. It has three main steps: identifying the defective gene, cloning the normal gene, and inserting the normal gene into target cells. There are two main approaches - ex vivo, where cells are removed and modified before being returned, and in vivo, where genes are directly inserted. Gene therapy aims to either augment gene function for loss of function mutations or inhibit gene expression for negative dominant mutations. While promising results have been seen for some diseases, risks still include immune reactions and insertional mutagenesis.
Gene therapy is a technique used to substitute defective genes that cause diseases with functional genes. It involves identifying the defective gene, extracting DNA, inserting a functional gene, and reinserting the modified DNA. There are two main types of gene therapy - somatic cell gene therapy, which treats only the individual and is not hereditary, and germline cell gene therapy, which could affect future generations but may be most effective. Gene therapy delivers the new genes via viral or bacterial vectors and holds promise to cure genetic diseases but also poses risks such as immune reactions or unintended effects.
Gene therapy is an experimental technique that uses viruses or nanotechnology to deliver working genes to replace defective genes that cause disease. It can be used to treat monogenic disorders caused by single gene defects as well as more complex disorders. There are three main types of gene therapy - germline gene therapy alters genes in egg and sperm cells and passes the changes to future generations; somatic gene therapy only alters genes in the patient's own body; and gene delivery methods like viruses must be used to safely introduce new genes. While gene therapy holds promise for treating many genetic diseases, there are also risks like immune reactions that require further research.
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
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 is an experimental technique that uses genes to treat or prevent disease. It works by inserting a normal gene to replace a defective gene that is causing disease. Researchers are studying gene therapy for diseases like cancer, HIV, hemophilia, blindness, and Parkinson's disease. There are two main types - germline gene therapy, which results in permanent changes that can be inherited, and somatic gene therapy, which only affects the patient. Gene delivery methods include viral vectors like retroviruses and adenoviruses, as well as non-viral methods using physical approaches or chemical carriers like liposomes and polymers. Some successful applications of gene therapy include treating blindness and reducing Parkinson's disease symptoms.
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 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.
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 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 therapy involves introducing genes into cells to treat or prevent disease. It works by delivering a therapeutic gene into a patient's target cells using a vector. This can replace an abnormal gene with a healthy one, inactivate a mutated gene, or introduce a new gene to fight disease. There are two main types - somatic gene therapy only affects treated cells while germline gene therapy results in permanent changes that can be inherited. Approaches include ex vivo gene therapy, which modifies cells outside the body before transplant, and in vivo gene therapy through direct delivery. While gene therapy holds promise to cure genetic diseases, it faces challenges like short-lived effects and safety risks.
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 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.
Advances in biochemistry and molecular biology have helped to understand the genetic basis of inherited diseases.
Gene therapy was once considered a fantasy (imaginary).
It was a dream of the researchers to replace the defective genes with good ones and cure the genetic disorders.
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 therapy aims to treat diseases by introducing normal genes into cells containing defective genes. The first approved gene therapy occurred in 1990 and treated ADA-SCID. There are two main types of gene therapy - germline modifies heritable genes while somatic only affects treated cells. Viral and non-viral vectors are used to deliver genes, with retroviruses and adenoviruses commonly used viral vectors. Recent advances include gene therapies reducing symptoms for blindness and Parkinson's disease.
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.
The document discusses gene therapy, which involves inserting normal genes into patients to replace abnormal genes that cause diseases. The first approved gene therapy experiment occurred in 1990 when a 4-year-old girl with severe combined immunodeficiency was treated. There are two main types of gene therapy - somatic cell gene therapy, which treats cells in the body but is not inherited, and germ line gene therapy, which treats eggs and sperm and can be inherited but has safety and ethical concerns. Viruses are commonly used as vectors to deliver therapeutic genes directly to tissues or cells can be removed and treated ex vivo before being returned to the body. While gene therapy holds promise, it also faces challenges and risks that require further research.
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 functional genes into patients to treat diseases. It has three main steps: identifying the defective gene, cloning the normal gene, and inserting the normal gene into target cells. There are two main approaches - ex vivo, where cells are removed and modified before being returned, and in vivo, where genes are directly inserted. Gene therapy aims to either augment gene function for loss of function mutations or inhibit gene expression for negative dominant mutations. While promising results have been seen for some diseases, risks still include immune reactions and insertional mutagenesis.
Gene therapy is a technique used to substitute defective genes that cause diseases with functional genes. It involves identifying the defective gene, extracting DNA, inserting a functional gene, and reinserting the modified DNA. There are two main types of gene therapy - somatic cell gene therapy, which treats only the individual and is not hereditary, and germline cell gene therapy, which could affect future generations but may be most effective. Gene therapy delivers the new genes via viral or bacterial vectors and holds promise to cure genetic diseases but also poses risks such as immune reactions or unintended effects.
Gene therapy is an experimental technique that uses viruses or nanotechnology to deliver working genes to replace defective genes that cause disease. It can be used to treat monogenic disorders caused by single gene defects as well as more complex disorders. There are three main types of gene therapy - germline gene therapy alters genes in egg and sperm cells and passes the changes to future generations; somatic gene therapy only alters genes in the patient's own body; and gene delivery methods like viruses must be used to safely introduce new genes. While gene therapy holds promise for treating many genetic diseases, there are also risks like immune reactions that require further research.
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
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 is an experimental technique that uses genes to treat or prevent disease. It works by inserting a normal gene to replace a defective gene that is causing disease. Researchers are studying gene therapy for diseases like cancer, HIV, hemophilia, blindness, and Parkinson's disease. There are two main types - germline gene therapy, which results in permanent changes that can be inherited, and somatic gene therapy, which only affects the patient. Gene delivery methods include viral vectors like retroviruses and adenoviruses, as well as non-viral methods using physical approaches or chemical carriers like liposomes and polymers. Some successful applications of gene therapy include treating blindness and reducing Parkinson's disease symptoms.
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 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.
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 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 therapy involves introducing genes into cells to treat or prevent disease. It works by delivering a therapeutic gene into a patient's target cells using a vector. This can replace an abnormal gene with a healthy one, inactivate a mutated gene, or introduce a new gene to fight disease. There are two main types - somatic gene therapy only affects treated cells while germline gene therapy results in permanent changes that can be inherited. Approaches include ex vivo gene therapy, which modifies cells outside the body before transplant, and in vivo gene therapy through direct delivery. While gene therapy holds promise to cure genetic diseases, it faces challenges like short-lived effects and safety risks.
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 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.
Advances in biochemistry and molecular biology have helped to understand the genetic basis of inherited diseases.
Gene therapy was once considered a fantasy (imaginary).
It was a dream of the researchers to replace the defective genes with good ones and cure the genetic disorders.
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 therapy aims to treat diseases by introducing normal genes into cells containing defective genes. The first approved gene therapy occurred in 1990 and treated ADA-SCID. There are two main types of gene therapy - germline modifies heritable genes while somatic only affects treated cells. Viral and non-viral vectors are used to deliver genes, with retroviruses and adenoviruses commonly used viral vectors. Recent advances include gene therapies reducing symptoms for blindness and Parkinson's disease.
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.
The document discusses gene therapy, which involves inserting normal genes into patients to replace abnormal genes that cause diseases. The first approved gene therapy experiment occurred in 1990 when a 4-year-old girl with severe combined immunodeficiency was treated. There are two main types of gene therapy - somatic cell gene therapy, which treats cells in the body but is not inherited, and germ line gene therapy, which treats eggs and sperm and can be inherited but has safety and ethical concerns. Viruses are commonly used as vectors to deliver therapeutic genes directly to tissues or cells can be removed and treated ex vivo before being returned to the body. While gene therapy holds promise, it also faces challenges and risks that require further research.
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.
The document discusses gene therapy and its potential to treat genetic diseases. It describes how gene therapy works by introducing functional genes into cells to replace defective genes causing disease. The first approved gene therapy treated a girl with ADA-SCID by inserting a functional ADA gene. While promising, gene therapy faces challenges like short-lived effects and safety issues that must still be addressed.
Gene therapy involves introducing genes into cells to treat disease. It works by correcting defective genes that cause diseases. The first approved gene therapy occurred in 1990 when a child with ADA-SCID was treated. There are two main types of gene therapy - somatic cell therapy targets body cells while germline therapy affects eggs and sperm. Significant challenges remain for gene therapy including developing safe methods for long-term effects and ensuring equitable access to new treatments. However, gene therapy has potential to cure many inherited diseases and revolutionize medicine.
This document provides an overview of gene therapy. It defines gene therapy as using genes or oligonucleotide sequences as therapeutic molecules to treat genetic defects. The document describes the types of gene therapy, strategies used, methods of delivery including ex vivo and in vivo approaches, target cells, vectors, advantages and disadvantages. It also discusses the current status of gene therapy and diseases where successful clinical trials have been reported.
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.
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.
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 and its potential to treat diseases. It provides an overview of genes and how mutations can cause disease. Gene therapy involves inserting functional genes into patients' cells to replace mutated genes that cause illness. Viruses called vectors are used to deliver therapeutic genes into target cells. Some common vector types discussed are retroviruses, adenoviruses, and adeno-associated viruses. Both ex vivo and in vivo gene therapy approaches are described. The document reviews the history of gene therapy and various clinical trials that have been conducted to treat diseases like cancer, cystic fibrosis, and immunodeficiencies.
Gene therapy and stem cells are discussed. [1] Gene therapy involves delivering genetic material into cells for therapeutic purposes using vectors. It aims to replace defective genes, enhance gene expression, or suppress harmful genes. [2] Stem cells are undifferentiated cells that can develop into specialized cell types and have self-renewal abilities. There are embryonic, adult tissue specific, and induced pluripotent stem cells. Gene therapy and stem cells offer potential treatments for diseases like cancer, genetic disorders, and tissue regeneration. However, safety and delivery challenges remain.
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.
This document provides an overview of gene therapy, including what it is, different types and approaches, vectors used, methods of delivery, advantages and disadvantages. Gene therapy involves inserting a normal gene to replace an abnormal gene responsible for a disease. It can be done via in vivo or ex vivo methods. Viral and non-viral vectors are used to deliver genes. While gene therapy holds promise to treat genetic diseases, it also faces challenges such as short-lived effects and safety issues.
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 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.
On January 25, 2022, Nature published an article listing seven technologies worthy of attention this year. Targeted genetic therapies was on the list. The remaining six technologies are: Fully finished genomes, Protein structure solutions, Quantum simulation, Precise genome manipulation, Spatial multi-omics), CRISPR-based diagnostics.
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.
Gene therapy involves inserting normal genes into patients to replace abnormal genes that cause disease. It is being studied for many diseases like immunodeficiencies, hemophilia, Parkinson's, and cancer. The first gene therapy occurred in 1990 and involved treating a genetic immune deficiency. While it offers potential cures, there are also risks and ethical concerns around its use.
The document discusses genes and genetic disorders. It defines a gene as the basic physical and functional unit of heredity, and notes that genes are segments of DNA that code for specific traits. Genetic disorders are caused by variations or mutations in genes. The document then discusses recent discoveries in epigenetics, gene marking, and gene therapy. Gene therapy involves introducing new genes into cells to treat diseases, either by replacing defective genes, inactivating abnormal genes, or introducing new genes to fight diseases. Various gene therapy approaches and vectors, including viral vectors like retroviruses and adenoviruses, are described.
These lecture slides, by Dr Sidra Arshad, offer a quick overview of the physiological basis of a normal electrocardiogram.
Learning objectives:
1. Define an electrocardiogram (ECG) and electrocardiography
2. Describe how dipoles generated by the heart produce the waveforms of the ECG
3. Describe the components of a normal electrocardiogram of a typical bipolar lead (limb II)
4. Differentiate between intervals and segments
5. Enlist some common indications for obtaining an ECG
6. Describe the flow of current around the heart during the cardiac cycle
7. Discuss the placement and polarity of the leads of electrocardiograph
8. Describe the normal electrocardiograms recorded from the limb leads and explain the physiological basis of the different records that are obtained
9. Define mean electrical vector (axis) of the heart and give the normal range
10. Define the mean QRS vector
11. Describe the axes of leads (hexagonal reference system)
12. Comprehend the vectorial analysis of the normal ECG
13. Determine the mean electrical axis of the ventricular QRS and appreciate the mean axis deviation
14. Explain the concepts of current of injury, J point, and their significance
Study Resources:
1. Chapter 11, Guyton and Hall Textbook of Medical Physiology, 14th edition
2. Chapter 9, Human Physiology - From Cells to Systems, Lauralee Sherwood, 9th edition
3. Chapter 29, Ganong’s Review of Medical Physiology, 26th edition
4. Electrocardiogram, StatPearls - https://www.ncbi.nlm.nih.gov/books/NBK549803/
5. ECG in Medical Practice by ABM Abdullah, 4th edition
6. Chapter 3, Cardiology Explained, https://www.ncbi.nlm.nih.gov/books/NBK2214/
7. ECG Basics, http://www.nataliescasebook.com/tag/e-c-g-basics
Cell Therapy Expansion and Challenges in Autoimmune DiseaseHealth Advances
There is increasing confidence that cell therapies will soon play a role in the treatment of autoimmune disorders, but the extent of this impact remains to be seen. Early readouts on autologous CAR-Ts in lupus are encouraging, but manufacturing and cost limitations are likely to restrict access to highly refractory patients. Allogeneic CAR-Ts have the potential to broaden access to earlier lines of treatment due to their inherent cost benefits, however they will need to demonstrate comparable or improved efficacy to established modalities.
In addition to infrastructure and capacity constraints, CAR-Ts face a very different risk-benefit dynamic in autoimmune compared to oncology, highlighting the need for tolerable therapies with low adverse event risk. CAR-NK and Treg-based therapies are also being developed in certain autoimmune disorders and may demonstrate favorable safety profiles. Several novel non-cell therapies such as bispecific antibodies, nanobodies, and RNAi drugs, may also offer future alternative competitive solutions with variable value propositions.
Widespread adoption of cell therapies will not only require strong efficacy and safety data, but also adapted pricing and access strategies. At oncology-based price points, CAR-Ts are unlikely to achieve broad market access in autoimmune disorders, with eligible patient populations that are potentially orders of magnitude greater than the number of currently addressable cancer patients. Developers have made strides towards reducing cell therapy COGS while improving manufacturing efficiency, but payors will inevitably restrict access until more sustainable pricing is achieved.
Despite these headwinds, industry leaders and investors remain confident that cell therapies are poised to address significant unmet need in patients suffering from autoimmune disorders. However, the extent of this impact on the treatment landscape remains to be seen, as the industry rapidly approaches an inflection point.
Rasamanikya is a excellent preparation in the field of Rasashastra, it is used in various Kushtha Roga, Shwasa, Vicharchika, Bhagandara, Vatarakta, and Phiranga Roga. In this article Preparation& Comparative analytical profile for both Formulationon i.e Rasamanikya prepared by Kushmanda swarasa & Churnodhaka Shodita Haratala. The study aims to provide insights into the comparative efficacy and analytical aspects of these formulations for enhanced therapeutic outcomes.
Adhd Medication Shortage Uk - trinexpharmacy.comreignlana06
The UK is currently facing a Adhd Medication Shortage Uk, which has left many patients and their families grappling with uncertainty and frustration. ADHD, or Attention Deficit Hyperactivity Disorder, is a chronic condition that requires consistent medication to manage effectively. This shortage has highlighted the critical role these medications play in the daily lives of those affected by ADHD. Contact : +1 (747) 209 – 3649 E-mail : sales@trinexpharmacy.com
Integrating Ayurveda into Parkinson’s Management: A Holistic ApproachAyurveda ForAll
Explore the benefits of combining Ayurveda with conventional Parkinson's treatments. Learn how a holistic approach can manage symptoms, enhance well-being, and balance body energies. Discover the steps to safely integrate Ayurvedic practices into your Parkinson’s care plan, including expert guidance on diet, herbal remedies, and lifestyle modifications.
TEST BANK For An Introduction to Brain and Behavior, 7th Edition by Bryan Kol...rightmanforbloodline
TEST BANK For An Introduction to Brain and Behavior, 7th Edition by Bryan Kolb, Ian Q. Whishaw, Verified Chapters 1 - 16, Complete Newest Versio
TEST BANK For An Introduction to Brain and Behavior, 7th Edition by Bryan Kolb, Ian Q. Whishaw, Verified Chapters 1 - 16, Complete Newest Version
TEST BANK For An Introduction to Brain and Behavior, 7th Edition by Bryan Kolb, Ian Q. Whishaw, Verified Chapters 1 - 16, Complete Newest Version
- 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
- Link to NephroTube website: www.NephroTube.com
- Link to NephroTube social media accounts: https://nephrotube.blogspot.com/p/join-nephrotube-on-social-media.html
1. GENE THERAPY
INTRODUCTION:-
The new human genome era is creating increasing possibilities for genomics-based
medicine called “personalized medicine.”1 Gene therapy, pharmacogenetics, and
pharmacogenomics are 3 major therapeutic interventions that take into account an individual’s
specifi c .genetic makeup. Genomics involves the study of genes and their surrounding DNA
sequences as well as the structure and function of the human genome.Genes are made up of a
chemical code (DNA) particular to each gene. Human genome discoveries have revealed that
approximately 25,000 genes reside in each individual’s human genome.
The code differs in sequence from gene to gene and directs the composition and production
of proteins that in turn make up living tissues and regulate all of the body’s functions. Genetic
disorders arise when an error in the complex, multistep process of replication and cell division
occurs. The error may be slight—perhaps just one unit of the code is misspelled, repeated, or
deleted—but its corresponding protein will be similarly improperly put together. When the
protein is essential enough, the error may lead to a sequence of events that can cause disability or
even deat Gene therapy interventions are being developed to treat three types of genetically
caused conditions. These are
Single – gene
Multi-factorial
Acquired genetic conditions.
DEFINITION OF GENE THERAPY
Gene therapy involves the correction of defective genes responsible for diseases such as
cancer gene therapy approach seeks to provide therapeutic benefit to a patient by introducing
normal genes into the patient’s cell nuclei to repair, enhance, replace, or compensate for an
altered gene
CLASSIFICATION OF GENETIC DISOREDDER BASED ON GENE INVOLVED
2. 1. Single gene disorders Mendelian genetic disorders and include such conditions as
cystic fibrosis, hemophilia, sickle-cell anemia, and Huntington’s disease
2. Multifactorial genetic disorders:-Conditions caused by a combination of genetic and
environmental influence.
3. Acquired genetic conditions are those that occur as a result of a viral infection such as
hepatitis or acquired immunodeficiency syndrome. In these conditions, the disorder is
caused by the new genetic information the virus carries into the host.
PRINCIPLES OF GENE THERAPY
Gene therapy approach seeks to provide therapeutic benefit to a patient by introducing
normal genes into the patient’s cell nuclei to repair, enhance, replace, or compensate for
an altered gene.
Gene therapy strategies under investigation include inserting a new functioning gene into
the cells of a patient to correct a genetic abnormalities or birth defect, thereby providing a
new function for the cell.
Switching the abnormal gene for a normal gene through a technique called homologous
recombination repairing the abnormal gene to return the gene to its normal function; and
regulating it.
Gene therapy offers the potential for treating many genetic disorders as well as cancer,
infectious diseases, and autoimmune disorders by genetically modifying cells in the
human body.
Current gene therapy initiatives are aimed at somatic cells, which are the non
reproductive cells of the body (eg skin, muscle, bone, and liver), somatic gene therapy,
can correct inherited genetic disorders and is limited to only one generation. Gene
therapy aimed at altering sperm and ova (reproductive cells) is called germ-line gene
therapy.
Enhancement gene therapy and eugenics are two other possible uses of gene therapy:-
Principle behind enhancement gene therapy is the placement of genes in an embryo or
offspring that would improve a societally desirable trait, such as decreased weight or
increased height.
Gene therapy used for eugenic purposes involves the introduction of specific genetic
traits into a population to develop “desirable” human attributes such as intelligence
USES OF GENE THERAPY :-
(l) Delivering the gene to the target tissue efficiently
(2) Sustaining long-term gene expression
(3) Ensuring that the gene transfer will not harm the patient in any way
(4) Transferring the corrected gene to non-dividing cells.
GENE TRANSFER
3. INVITRO:- It is also called ex vivo. in vitro approach requires that the defective cells or
cells of interest be removed from the patient fi rst. The corrected or marker gene is then
inserted into the cells, and the altered cells are returned to the individual. The cells most
commonly used for this approach include lymphocytes, skin fibroblasts, and tumor and
bone marrow cells. These cells are readily accessible, amenable to manipulation, and able
to survive for long periods of time following reinfusion.
INVIVO:- In vivo gene transfer involves the direct delivery of therapeutic genes to target
body cells. In vivo approaches to gene therapy have been used in clinical trials for cystic
fibrosis (CF), muscular dystrophy, melanoma, and heart, lung, and metabolic conditions.
With in vivo gene transfer, naked DNA can be delivered without the use of needles, using
a gene gun or jet gun. Both methods use either high pressure helium or liquid to deliver
the DNA to interstitial places.
VECTORS FOR GENE TRANSFER
VIRAL VECTOR:- gene therapy uses viral vectors to deliver the therapeutic gene to the target
tissue. All viruses used have been disabled of any pathogenic effects by removing the genes
required for replication of the virus and replacing them with therapeutic genes and selection
markers. The use of viruses is a potentially powerful technique because many have evolved
specific mechanisms for delivering DNA to cells.
I. RETROVIRAL VECTORS:-A retrovirus is composed of RNA that can insert itself
readily into dividing cells. Retroviruses are considered the most promising gene
transfer vehicle. These RNA viruses are able to carry out effi cient gene transfer into
many types of cells and can integrate into the host cell genes with stability.
Advantage
The therapeutic gene carried into the cell by the retrovirus will be inherited by all
future generations of the cell and will provide the possibility of long-term gene
expression
Disadvantage:-
4. The insertion of the retrovirus will disrupt normal genes essential for proper cell
function, leading to harmful physiological effects that favor cancer development.
II. LENTIVIRAL VECTORS:- Lentiviruses, which belong to the retrovirus family, are
now being used in gene therapy because they can infect both dividing and
nondividing cells and have the ability to provide long-term and stable gene
expression. Human immune deficiency virus (HIV) is the most well-known lentivirus
used for gene transfer in vivo.
Advantage
The use of lentiviruses such as HIV as a vector for gene therapy may not cause problems,
such as cancer, that have arisen with retroviral vectors
III. ADENOVIRAL VECTORS:-Adenoviruses are a family of viruses that cause benign
respiratory tract, intestinal, and eye infections in humans. They also hav the capacity
to infect both dividing and nondividing cells, making them useful for gene therapy.
Advantages
Adenoviruses are large and can hold large segments of therapeutic DNA.
They can also be produced in large amounts in culture.
They have been the vectors of choice for many protocols designed to treat the pulmonary
complications of cystic fibrosis as well as for a variety of clinical protocols to treat
cancer.
In contrast to retroviruses, which contain RNA, adenoviruses contain DNA and thus do
not integrate into host DNA but instead replicate themselves outside of the nucleus of the
host cell. Because of this limited integration, expression of the therapeutic gene is short-
lived, and regular reapplication of gene therapy using adenovirus vectors is necessary.
The potential usefulness of adenoviral vectors stems from the fact that they do not require
actively dividing cells to introduce their therapeutic gene.
Disadvantage:-
5. Adenoviruses, however, are a common cause of upper respiratory tract infections in
humans. As a result, unfortunately, most of the human population may experience an
active immune response to antibodies from a previous infection, which could reduce the
effectiveness of gene therapy using this vector.
Another potential concern with using adenoviral vectors is that the integrated gene may
not lead to uniform correction of the gene defect, because it may not remain active in the
host cell. Other viral vectors that may potentially enhance the delivery of therapeutic
genes are therefore being explored.
IV. Newer adenoviral vectors:- The adeno-associated virus (AAV) is one of the newer
viral vectors under investigation; it is a simple, nonpathogenic virus composed of a
single strand of DNA. In order to replicate, AAV needs additional genes. In the past,
a helper virus, usually adenovirus or herpes simplex virus, served this purpose. The
AAV virus can infect a variety of types of cells, and although it appears to integrate
in a nonspecifi c manner, it has been shown to integrate preferentially into
chromosome 19. AAV gene therapy has been investigated for cysticfi brosis and for
factor IX hemophilia.Other viruses that are being considered and developed for use as
vectors for gene therapy are the herpes simplex virus, which infects cells of the
nervous system, and the vaccinia virus. These viral vector systems produce a transient
response, and many people have an immunity to components of the virus from being
infected previously.
CLINICAL PROTOCOLS FOR GENE THERAPY
Clinical protocol for gene therapy was initiated in 1990.
Two girls with ADA deficiency, a rare genetic condition that produces severe immune -
defi ciency in children, were injected with white blood cells carrying a therapeutic gene.
The clinical protocol called for inserting the ADA gene into T lymphocytes.
ADA is an enzyme needed for normal immune system functioning. It prevents the
buildup of deoxy-adenosine, a metabolic product that becomes toxic to immune cells,
especially lymphocytes, when present in high concentrations.
ADA deficiency accounts for 25% of cases of severe combined immunodeficiency
disease (SCID)
Treatment for ADA deficiency has also demonstrated the Benefits and risks of gene
therapy. In 2003,
French researchers reported that two children with SCID who had been treated with
retroviral gene therapy had developed a leukemia-like disorder 30 months after a single
gene therapy treatment. In response to this discovery, the FDA put a hold on clinical
trials that used retroviral vectors to insert the defective gene into hematopoietic cells.
The American Society of Gene Therapy conducted its own investigation to determine
why this occurred only in patients with SCID, and not in other trials with retroviruses.
Researchers have since discovered that the retrovirus used (Moloney-murine leukemia
6. virus, or Mo-MuLV) inserted the therapeutic genes next to a gene known to promote
blood cancer.
Insertional mutagenesis is a recognized complication of retroviral gene transfer attempts
because gene integration occurs randomly. With Mo-MuLV, this appears to occur at the
beginning of the gene, affecting how it works.
GENE THERAPY REGULATION
Guidelines for clinical gene therapy protocols were established by the National
Institutes of Health (NIH) in the document Points to Consider in the Design and
Submission of Human Somatic Cell Gene Therapy Protocols in 6 parts as given
below:-
Concern for the clinical benefit of all persons receiving
gene therapy
Assurance of informed consent
Fair selection of persons for gene therapy–research Protocols
Attention to the need for bio-safety protocols
Public involvement in genetic research policy
Attention to long-term consequences of genetic research.
The Human Gene Therapy Research Subcommittee and the Recombinant DNA
Advisory Committee (RAC) then review the protocol.
Above mentioned two committees serve in an advisory capacity to the director of
the NIH, who approves all gene transfer and gene-therapy proposals.
The FDA addresses the scientific methodology and preclinical safety testing and
has created a set of guidelines for the initiation of gene therapy.The FDA’s
guidelines are separate from the Human Genome Research Subcommittee and
RAC guidelines and address the characteristics, production, and certification of
the biological substances being used for gene transfer.
The “Points to Consider” document is updated regularly and is found as an
appendix of the NIH’s guidelines on recombinant DNA research.
CANCER GENE THERAPY
Introduction to oncogenes :-
Proto-oncogenes are normal cellular genes that are essential for cellular growth and
development. Oncogenes stimulate neoplastic growth and are activated by proto-oncogenes that
encode a growth factor or another protein and disturb normal cell development and regulation.
Antioncogenes are those genes that block the action of growth-inducing proteins. These genes
are also called tumor-suppressor genes to denote their ability to block the action of oncogenes.
When functioning normally, tumor-suppressor genes and proto-oncogenes work together to
enable the body to perform vital functions, such as replacing dead cells and repairing defective
ones.
Definition:- cancer gene therapy is defined as inhibiting oncogene function and restoring
tumor-suppressor function.
7. Types of gene transfer in clinical cancer gene therapy:-
o Gene marking
o Gene therapy
o Gene marking :- it involves labeling cells for future identifi cation. A gene that has been
genetically marked is introduced into cells, most commonly using a retrovirus as a vector
for the desired gene. Gene marking studies have been used in the treatment of melanoma,
leukemia, neuroblastoma, and stem cell transplantation.
o Gene therapy:- it involve modifi cation of the content or expression of altered genes in
somatic cells by transferring the functional or enhanced genes.
PROTOCOL OF CANCER GENE THERAPY:-
1.Tumor -directed approach:- in this approach therapeutic gene is introduced into tumor cells
to destroy them.
I. “Suicide gene” therapy protocols: suicide gene is that which produces an enzyme
whose activity converts a nontoxic prodrug to its toxic form. The gene transfer is
targeted to tumor cells to make them susceptible to an agent that does not cause harm to
normal cells but kills malignant cells. The suicide gene is toxic to dividing cells only,
thus sparing the normal cells and non dividing tumor cells. A new approach to suicide-
gene therapy uses ultrasoundand nano/microbubbles (NBs) to deliver exogenous
molecules noninvasively into a specifi c target site. Examples :- herpes simplex virus
thymidine kinase gene (HSV-TK) is the one most commonly used. Retroviral vectors
transfer genes to actively dividing cells, making this type of gene therapy well suited for
the treatment of brain tumors.
II. Tumor-suppressor gene therapy: The tumor-suppressor gene, P53, is found in
approximately 50% of all cancers. A normal copy of the gene has been introduced to
restore its function in patients with lung cancer, colorectal cancer, breast cancer, as well
as many others.
III.Antisense oligonucleotides: Genetic therapies for cancer treatment are being developed
that specifi cally target DNA and RNA. The use of specifi c segments of DNA—
antisense oligonucleotides—is one example of this new methodology. Antisense
oligonucleotides are nucleic binding agents. They are short strands of nucleotides that
predictably combine with other nucleotides. This property allows for the design of a
treatment drug that can recognize a unique site on a specifi c gene. Oligonucleotides can
be inserted into cells to interfere with the translation of RNA into an oncogene protein.
When transferred into patients, they prevent the oncogene’s RNA message from being
translated into a functional oncogene protein. This approach is being used in clinical trials
of antiepidermal growth factor receptor in the treatment of head and neck squamous cell
carcinoma.
IV. Oncolytic viral gene therapy: Oncolytic viruses reproduce in tumor cells, causing their
lyses. These viruses can be used to treat cancers with a defective p53 pathway. In normal
8. cells, p53 is destroyed by adenoviruses, producing a protein called E1B 55K. This protein
binds with p53 and, in synchrony with another viral protein, directs p53 for destruction.
Researchers have been able to modify a strain of adenovirus so that it does not encode
E1B 55K and lose its ability to destroy p53. Normally, p53 activity causes a cascade of
events that lead to the death of the affected cell, and viral replication cannot be
established. In cancer cells that lack p53, however, the normal mechanism that leads to
the death of the infected cells is abolished so that only the modified adenovirus can
replicate and kill tumor cells. Used to treat head and neck cancer and, in some clinical
trials, is being combined with chemotherapeutic agents.
2(A) Active immunotherapy
I. Tumor-infi ltrating lymphocytes: Host immunological responses can be used to alter the
natural course of some cancers, especially malignant melanoma. Tumor-infiltrating
lymphocytes (TILs) that are able to mediate tumor regression in patients with melanoma
have been identified. Administration of an epitope in conjunction with intravenous high-
dose interleukin-2 (IL-2) has been shown to mediate regression in patients with malignant
melanoma.
II. Cytokine genes: Cytokines are molecules that enhance the body’s immune response to
tumor antigens. Cytokine genes are being used to augment the body’s ability to mount an
immune response to tumor cells. Two of the most commonly used cytokine genes are IL-
2 and tumor necrosis factor. These cytokine genes are modified so that they will not
proliferate but can still support expression of the introduced gene products. The modifi ed
cells are then injected into the patient via subcutaneous, intradermal, or intramuscular
routes.
III. DNA vaccines: DNA vaccines that contain tumor associated–antigen genes or cytokine
genes are now being used because the pure DNA encodes only the tumor associated–
antigen gene. DNA vaccines are usually administered intramuscularly, alone or in
combination with cytokine genes.34 Example : Metastatic renal cell carcinoma and
prostate is one cancer that has demonstrated responsiveness to immunotherapeutic
intervention.
2(B) Adoptive immunotherapy:-
Adoptive immunotherapy is a form of gene-transfer therapy in which the patient’s own
lymphocytes (peripheral blood or from TILs) are modifi ed outside of the body with genes
that enhance their antitumor activity. The modifi ed genes are then reinfused back into the
same patient.
I. Chimeric receptors:- it consist of two different molecules brought together to form one
functional molecule—an extracellular antibody molecule linked to intracellular signal
domains of T-cell receptors. In this type of therapy, the patient’s autologous T cells serve
as the vehicle for inserted chimeric antibody molecules. The inserted antibody molecules
have specifi city for a tumor antigen. When transferred into patients, the modified T cells
are redirected to recognize the tumor by virtue of the specific antibody on its surface.
Once the tumor cells are engaged, the T cells are activated via the signaling chain and
9. mediate antitumor activity. This approach is being used in the treatment of patients with
neuroblastoma, kidney cancer, and metastatic ovarian cancer.34,40
II. Tumor-specifi c T cells: Adoptive immunotherapy using transduction of the IL-2 gene
into antitumor T lymphocytes. Transduction of an IL-2 gene into a TIL has been used
with some success in the treatment of patients with melanoma and renal cell carcinoma.
PHARMACOGENETICS AND PHARMACOGENOMICS: GENE-BASED
TREATMENT OF CANCER
Pharmacogenomics :- comes from the terms pharmacology and genomics and is the intersection
between pharmaceuticals and genomics. Pharmacogenomics leads to the development of drugs
that can be adapted to each person’s specifi c genetic make-up.
Examples :-
Genetic polymorphism of thiopurine methyltransferase (TPMT). TPMT has been
associated with altered drug metabolism and increased risk for severe toxicity from the
anticancer agent 6-mercaptopurine.
CURRENT CHALANGES FOR GENE THERAPY
Current viral and nonviral vectors do not yet provide a completely satisfactory means of
propagating the therapeutic genes in proliferating cells. To increase the possibilities of
success, researchers are investigating the introduction of a 47th chromosome (artificial
chromosome) into target cells. This method does not affect the working of the other 46
chromosomes or cause mutations. The artifi cial chromosome can serve as a natural
human vector for therapeutic genes. Nonviral gene therapy methods offer the potential
for therapeutic interventions that may be acceptable to physicians and patients and offer
safety and efficiency similar to those of conventional therapeutic modalities.
FUTURE APPROACHES TO GENE THERAPY:-
A. RNA interference (RNAi):- RNAi is being used to block the expression of a specifi c
gene using this mechanism as a technique for exploring gene function and for treating
disease. Short interfering RNAs (siRNAs) are being used to avoid the problems with long
double stranded RNA molecules that cause the interferon response in some cell types.
There are currently more than 10 clinical trials using this gene therapy technique.
Pharmaceutical companies are also developing RNAi-based treatments for diseases
including
B. hepatitis C, age-related macular degeneration, asthma, and Huntington Disease.
C. Nanotechnology is being applied in cancer diagnostics and treatments. Nanotechnology-
based therapeutics approved for cancer treatment include Doxil, a liposome preparation
of doxorubicin, and Abraxane, which is paclitaxel in nanoparticle formulation. Using
nanoparticles, chemotherapy drugs can now be delivered directly to tumor cells and then
send a signal after the cancer cells are destroyed.
ETHICAL, SOCIAL, AND LEGAL ISSUES IN GENE THERAPY
10. Two serious and life-threatening genetic disorders—ADA defi ciency and alpha-
thalassemia—are under consideration for fetal gene therapy. Current issues being debated
include whether it is better to treat a disease for which backup therapies exist, as is the
case for ADA defi ciency, or to go forward with gene treatment for alpha-thalassemia, a
blood disorder that is often fatal to fetuses.
Genetic testing and gene therapies reveal information about individuals and family
members. This information has the potential to label currently healthy individuals as
being “at risk.” As genetic testing and therapeutics become more common, personal, and
family genetic information may inadvertently become public.
Cost of genetic therapy.
Accessibility of genetic services
ROLE AND RESPOSIBITIES OF ONCOLOGY NURSES IN GENE THERAPY AND
ITS CLINICAL IMPLICATION:-
Direct Caregiver
• Provides anticipatory guidance
• Assures informed decision making/consent
• Develops treatment and management plans
• Administers gene therapy
• Observes patients for expected and unexpected side effectsof treatment, including
psychosocial and emotional response
• Participates in developing long-term follow-up plans
• Assures coordination and collaboration of care with allhealthcare providers involved in
patient/family care before,during, and after gene therapy
Educator
• Serves as an information source to patients, families, and the public
• Provides relevant, accurate, and understandable information to patients, in both written
and verbal forms
• Assures that all patient/family questions are answered
Advocate
• Assures privacy and confi dentiality of genetic information
• Protects against discrimination
• Advocates for fair and equitable use of gene therapies for all populations
• Promotes public understanding of somatic gene therapy
General Services Provider
• Gathers relevant family history information
• Identifi es individuals and families in need of further genetic education and counseling
• Assesses psychosocial, ethnoculture, and educational background
• Provides psychosocial support in follow-up to genetic counseling
Research Investigator
• Participates in or conducts clinical-research trials in gene therapy
• Serves as a preceptor to other nurses
11. • Develops research protocols that will address patient/ family response and adaptation to
genetic information, including gene therapy
Bibliography
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GENE Therapy