gene therapy
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The document discusses gene therapy, which involves introducing genes into cells to treat or cure diseases. It describes two main types of gene therapy - in vivo, which delivers genes directly into a patient's body, and ex vivo, which modifies cells in culture before transplanting them into patients. Viral and non-viral vectors are used to deliver therapeutic genes. The document outlines several applications of gene therapy, such as for genetic disorders, cancer, and autoimmune diseases. However, it notes that gene therapy faces challenges and high costs that limit widespread accessibility currently.
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Human Genome Engineering
The document discusses human genome engineering and the creation of designer babies. It begins by introducing the topic and some key terms like pre-implantation genetic diagnosis and germline engineering. It then covers current methods used like CRISPR/Cas9 gene editing and mitochondria replacement therapy. The document addresses both the benefits of reducing genetic diseases and longer lifespans but also the ethical considerations regarding safety and genetic enhancement. It concludes by discussing some recent successes in the field while noting more research is still needed to fully assess safety.
Gene therapy ppt
This presentation provides an overview of gene therapy. It introduces gene therapy as a technique for introducing genes into cells to prevent or cure diseases by correcting defective genes. The first approved gene therapy experiment occurred in 1990. There are two main types of gene therapy: somatic cell gene therapy, which transfers genes into somatic cells and is not inherited, and germline gene therapy, which transfers genes into germ cells and can be inherited. Gene delivery can be done in vivo or ex vivo using viral or non-viral vectors. While still facing challenges, gene therapy has potential to cure inherited diseases and possibly cancers and heart disease in the future.
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.
Gene therapy
This document discusses gene therapy and genetic engineering. It distinguishes between gene therapy, which aims to treat disease by replacing mutated genes, and genetic enhancement, which aims to modify humans in a way that goes beyond curing illness or disability. While gene therapy seeks to cure disease and is considered acceptable, genetic enhancement that modifies humans to make them "super human" raises ethical issues and is generally not supported by the Catholic Church. The key distinction is that gene therapy should be therapeutic and only done to promote health, while genetic enhancement goes beyond therapy and could worsen conditions or unfairly advantage some individuals.
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,
Jmp gene therapy_intro
Genetic disorders can be caused by abnormalities in chromosomes or single genes. Chromosomal disorders occur when there are errors in chromosome number or structure during meiosis, leading to conditions like Down syndrome. Single gene disorders are caused by mutations in genes, and result in diseases like cystic fibrosis, sickle cell anemia, and Huntington's disease. Gene therapy aims to treat genetic disorders by inserting a normal gene into cells to replace the mutated version, using viral vectors to deliver the healthy gene. While some successes have been seen, challenges remain in developing safe and effective vector delivery systems.
GENE THERAPY.1.pptx
Above slides consist of detailed information of gene therapy,including types, methods, future needs along with advantages and disadvantages
Gene therapy - current status and future perspective
The document discusses gene therapy, including its types, steps involved, applications, current status, limitations, and future perspective. It provides details on gene therapy techniques like gene augmentation and gene inhibition. Key points covered include current clinical trials focusing on cancer and monogenic diseases, the use of viral vectors, and challenges relating to funding, ethics, and ensuring safe and effective minimum dosages. The future of gene therapy is seen as promising for treating many currently incurable conditions if optimization and accessibility issues can be addressed.
Recommended
Human Genome Engineering
The document discusses human genome engineering and the creation of designer babies. It begins by introducing the topic and some key terms like pre-implantation genetic diagnosis and germline engineering. It then covers current methods used like CRISPR/Cas9 gene editing and mitochondria replacement therapy. The document addresses both the benefits of reducing genetic diseases and longer lifespans but also the ethical considerations regarding safety and genetic enhancement. It concludes by discussing some recent successes in the field while noting more research is still needed to fully assess safety.
Gene therapy ppt
This presentation provides an overview of gene therapy. It introduces gene therapy as a technique for introducing genes into cells to prevent or cure diseases by correcting defective genes. The first approved gene therapy experiment occurred in 1990. There are two main types of gene therapy: somatic cell gene therapy, which transfers genes into somatic cells and is not inherited, and germline gene therapy, which transfers genes into germ cells and can be inherited. Gene delivery can be done in vivo or ex vivo using viral or non-viral vectors. While still facing challenges, gene therapy has potential to cure inherited diseases and possibly cancers and heart disease in the future.
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.
Gene therapy
This document discusses gene therapy and genetic engineering. It distinguishes between gene therapy, which aims to treat disease by replacing mutated genes, and genetic enhancement, which aims to modify humans in a way that goes beyond curing illness or disability. While gene therapy seeks to cure disease and is considered acceptable, genetic enhancement that modifies humans to make them "super human" raises ethical issues and is generally not supported by the Catholic Church. The key distinction is that gene therapy should be therapeutic and only done to promote health, while genetic enhancement goes beyond therapy and could worsen conditions or unfairly advantage some individuals.
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,
Jmp gene therapy_intro
Genetic disorders can be caused by abnormalities in chromosomes or single genes. Chromosomal disorders occur when there are errors in chromosome number or structure during meiosis, leading to conditions like Down syndrome. Single gene disorders are caused by mutations in genes, and result in diseases like cystic fibrosis, sickle cell anemia, and Huntington's disease. Gene therapy aims to treat genetic disorders by inserting a normal gene into cells to replace the mutated version, using viral vectors to deliver the healthy gene. While some successes have been seen, challenges remain in developing safe and effective vector delivery systems.
GENE THERAPY.1.pptx
Above slides consist of detailed information of gene therapy,including types, methods, future needs along with advantages and disadvantages
Gene therapy - current status and future perspective
The document discusses gene therapy, including its types, steps involved, applications, current status, limitations, and future perspective. It provides details on gene therapy techniques like gene augmentation and gene inhibition. Key points covered include current clinical trials focusing on cancer and monogenic diseases, the use of viral vectors, and challenges relating to funding, ethics, and ensuring safe and effective minimum dosages. The future of gene therapy is seen as promising for treating many currently incurable conditions if optimization and accessibility issues can be addressed.
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The document discusses gene therapy, including:
1. Defining genes and discussing early milestones in gene isolation and engineering.
2. Explaining the goal of gene therapy to introduce normal genes to compensate for defective genes.
3. Describing various methods of gene delivery including viral and non-viral vectors.
4. Discussing challenges in targeting specific cells/tissues and ensuring safe expression levels.
Gene therapy
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.
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Gene therapy involves inserting normal genes into cells to treat genetic diseases. There are two main methods - using viruses as vectors to deliver genes (viral vectors) or delivering naked DNA. Researchers are studying gene therapy for diseases like cancer, HIV, hemophilia and more. Some successes include treating blindness caused by a genetic defect using a virus to deliver a healthy gene to the eyes. Gene therapy has also reduced Parkinson's disease symptoms by delivering a gene to the brain to increase inhibitory neurotransmitters. While offering hope, gene therapy also faces challenges in terms of safety, cost and ethical issues.
Basics of gene therapy
This document discusses gene therapy, including its history, mechanisms, applications, and challenges. It begins by defining genes and their role in hereditary traits. It then explains how gene therapy works by inserting genes into cells to treat genetic diseases. Various gene therapy methods are outlined such as ex vivo therapy using cultured cells and in vivo direct delivery. Challenges including short duration of effects, immune responses, and safety issues from viral vectors are also summarized.
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Gene therapy
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
Gene therapy involves inserting a normal gene into cells to replace a mutated gene that causes disease. There are two main types - germline gene therapy affects reproductive cells and would alter the genes passed to future generations, while somatic gene therapy only affects the patient's body cells. Vectors like viruses are used to deliver the normal gene, but there are risks like unwanted immune reactions, infecting the wrong cells, or the vector virus recovering its ability to cause disease. While gene therapy holds promise, there remain technical limitations that must still be addressed.
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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.
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Gene Therapy.pdf
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.
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Gene therapy(dr ravindra daggupati)
gene therapy,applications of gene therapy,gene therapy in specific to otorhinolaryngology(ENT),types of gene therapy,gene therapy techniques
Dev gene therapy
The document discusses gene therapy, including:
1. Defining genes and discussing early milestones in gene isolation and engineering.
2. Explaining the goal of gene therapy to introduce normal genes to compensate for defective genes.
3. Describing various methods of gene delivery including viral and non-viral vectors.
4. Discussing challenges in targeting specific cells/tissues and ensuring safe expression levels.
Gene therapy
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.
Advances in Gene Therapy: Eyal Grunebaum (The Hospital for Sick Children)
Advances in Gene Therapy: Eyal Grunebaum (The Hospital for Sick Children)Canadian Organization for Rare Disorders
This document discusses advances in gene therapy. It begins by explaining what gene therapy is and provides examples of how it has been used to treat children with severe immune deficiencies who previously had to live in protective bubbles. While bone marrow transplants can cure these conditions, they often fail due to graft-versus-host disease. Gene therapy offers advantages over bone marrow transplants by using a patient's own cells with corrected genes, avoiding this complication. The document outlines the process of doing gene therapy outside the body by inserting genes into viruses to deliver them to patients' cells. It notes some of the early successes and challenges of gene therapy clinical trials.Gene therapy
Gene therapy involves inserting normal genes into cells to treat genetic diseases. There are two main methods - using viruses as vectors to deliver genes (viral vectors) or delivering naked DNA. Researchers are studying gene therapy for diseases like cancer, HIV, hemophilia and more. Some successes include treating blindness caused by a genetic defect using a virus to deliver a healthy gene to the eyes. Gene therapy has also reduced Parkinson's disease symptoms by delivering a gene to the brain to increase inhibitory neurotransmitters. While offering hope, gene therapy also faces challenges in terms of safety, cost and ethical issues.
Basics of gene therapy
This document discusses gene therapy, including its history, mechanisms, applications, and challenges. It begins by defining genes and their role in hereditary traits. It then explains how gene therapy works by inserting genes into cells to treat genetic diseases. Various gene therapy methods are outlined such as ex vivo therapy using cultured cells and in vivo direct delivery. Challenges including short duration of effects, immune responses, and safety issues from viral vectors are also summarized.
Gene therapy
gene therapy (introduction, types with examples, advantages, and disadvantages also include limitation of gene therapy.
Gene therapy
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
Gene therapy involves inserting a normal gene into cells to replace a mutated gene that causes disease. There are two main types - germline gene therapy affects reproductive cells and would alter the genes passed to future generations, while somatic gene therapy only affects the patient's body cells. Vectors like viruses are used to deliver the normal gene, but there are risks like unwanted immune reactions, infecting the wrong cells, or the vector virus recovering its ability to cause disease. While gene therapy holds promise, there remain technical limitations that must still be addressed.
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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.
Genome Editing & Gene Therapy by Eric Kelsic
Slides from the Genome editing & gene therapy Impact.tech seminar, hosted by Eric Kelsic on June 11th, 2019.
The seminar covers the experiments and inventions that led to the development of genome editing technologies. These inventions were derived from life itself: isolated from natural organisms and adapted for scientific and therapeutic goals. You will learn the history of how genome engineering tools, including CRISPR, and delivery technology, including AAV capsids, were created in their modern form. The seminar explores how genome editing and gene therapy technologies are giving individuals control over their own genomes, focusing on the treatment of genetic diseases. It will describe major companies and emerging trends in the gene therapy industry. Finally, the seminar will discuss how and where new discoveries, including accelerated algorithms for genetic engineering, will lead us in the near and distant future.
Eric Kelsic, PhD, is the founder and CEO of Dyno Therapeutics, a VC-backed biotech located in Cambridge, Massachusetts. Dyno is leading a machine learning revolution to develop enhanced capsid proteins that enable new gene and genome editing therapies. Eric co-developed the technology underlying Dyno’s machine-guided protein engineering platform as a Staff Scientist in George Church’s lab at the Wyss Institute of Harvard Medical School. He holds a PhD in Systems Biology from Harvard University and a BS in Physics from Caltech.
Gene therapy
Gene therapy is the science of making specific changes to the human genome to improve it or achieve therapeutic effects for gene-related diseases. This document discusses gene therapy in three sentences: it defines gene therapy, describes the three main types (germline, somatic, vaccination), and explains that somatic gene therapy aims to treat body cells without affecting future generations. The document then discusses the process of gene therapy including identifying defective genes, creating a replacement gene, and delivering the new gene into the body.
Genetic diagnosis by whole exome capture and massively parallel DNA sequencing
This document outlines a new genetic diagnosis protocol using whole exome capture and massively parallel DNA sequencing. The protocol allows for decreased DNA sequencing and cost while increasing detection of heterozygous bases and read depth. It was used to diagnose a patient with congenital chloride diarrhea through an unexpected genetic finding that confirmed the clinical diagnosis. The protocol has implications for discovering disease genes involved in Mendelian traits through identifying de novo mutations or homozygous segments, and for complex traits by applying selective sequencing of promising genes identified through initial sequencing of subjects.
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Gene therapy involves inserting genes into cells to treat disease. It was first tested in animals and then used in humans. The first approved gene therapy experiment occurred in 1990 and treated a child named Ashanti DeSilva for ADA-SCID. There are two main types of gene therapy - somatic cell gene therapy, which targets non-reproductive cells and is preferred since genetic changes are not passed to offspring, and germ line gene therapy, which targets reproductive cells. A common method is ex vivo gene therapy, which involves removing cells like bone marrow, modifying them in a lab, and returning them to the patient.
AGCT - cell gene therapy for HIV cure
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Genetic engineering of humans
This document discusses genetic engineering of humans through various methods such as germline genetic modification and somatic genetic modification. It notes both the promising applications for improving health through gene therapy but also the ethical concerns regarding enhancing traits and the possibility of genetic inequality. Specific examples discussed include using CRISPR to genetically modify human embryos to potentially treat disease as well as concerns about "designer babies". The document examines issues around testing for non-disease traits, gene doping in athletes, and the potential benefits and risks of human gene editing technologies.
Genetics, the pillars for new discoveries
The document discusses two recent studies on genetics and genetic engineering. The first study developed a new type of restriction enzyme that recognizes longer DNA cutting sites, making it more specific. The second study identified a rare form of Congenital Adrenal Hyperplasia caused by an 11 beta hydroxilase deficiency linked to consanguineous marriages in some regions. Knowing the complete genetic profile of diseases can help develop improved treatments. A student comments that both studies will aid genetic research and modifying DNA to treat diseases.
iMGE Test™
The document discusses a genetic test called iMGE Test that analyzes miscarriage material to identify chromosomal defects that may cause pregnancy loss. The test uses next generation sequencing to analyze all chromosomes simultaneously and does not require cell culture, providing results for over 95% of cases. Identifying genetic causes can help guide family planning decisions and determine if preimplantation genetic diagnosis would be beneficial for couples seeking to avoid miscarriage in future pregnancies. The test is recommended after miscarriage for women over 35, those with a family history of genetic defects, or couples with a history of infertility or recurrent miscarriage.
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Advances in Gene Therapy: Eyal Grunebaum (The Hospital for Sick Children)
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Genetic diagnosis by whole exome capture and massively parallel DNA sequencing
Genetic diagnosis by whole exome capture and massively parallel DNA sequencing
Non-Viral Φc31 Integrase Mediated In Vivo Gene Delivery to the Adult Murine K...
Non-Viral Φc31 Integrase Mediated In Vivo Gene Delivery to the Adult Murine K...
Similar to gene therapy
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.
Gene Therapy.pdf
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.
Gene therapy
This document provides an overview of gene therapy, including its types, approaches, vectors used, delivery methods, applications, advantages, and disadvantages. Gene therapy involves introducing normal genes into cells to replace missing or defective genes and correct genetic disorders. The first approved gene therapy experiment treated a child for ADA-SCID. Somatic and germline gene therapy are the two main types. Vectors deliver therapeutic genes and include viral vectors like retroviruses and lentiviruses and non-viral methods. Applications show promise for diseases like cancer, cystic fibrosis, hemophilia, and neurological disorders. However, risks include toxicity, immune responses, and unpredictable effects that require further research.
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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.
Gene therapy
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.
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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.
Gene therapy
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 ppt
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.
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.
GENE THERAPY
This document provides an overview of gene therapy, including types, approaches, vectors used, methods of delivery, advantages, disadvantages, applications, and recent advances. It discusses somatic cell gene therapy, which aims to correct genetic defects in non-reproductive cells, and germline gene therapy, which could pass alterations to future generations but poses more risks. Ex vivo and in vivo gene therapy approaches are described. Viral and non-viral vectors as well as various delivery methods are outlined. Some applications including cystic fibrosis and cancer are highlighted. Risks and ethical considerations are also mentioned.
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 based and cell based therapy in clinical medicine converted
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Nucleic acid based therapeutic delivery system
Myself Omkar Tipugade , PG Student of Department of Pharmaceutics. today I will discus on the topic Gene Therapy . In that we discus about the method for gene therapy & its application for disease treatment.
Gene therapy types advantags disadvantages
Gene therapy involves inserting normal genes into patients to replace abnormal genes responsible for disease. There are two main types of gene therapy: ex vivo involves removing cells, modifying them, and reinserting them; in vivo delivers genes directly into target tissues. Viral and non-viral vectors are used to deliver genes. While gene therapy has potential to treat many diseases, it also has disadvantages like short-term effects and safety risks that must still be addressed.
Genetherapypresentation 120220051948-phpapp02
Gene therapy is an experimental technique that aims to correct defective genes responsible for disease development. It works by inserting a normal gene to replace an abnormal one. Researchers are studying gene therapy for diseases like SCID, hemophilia, Parkinson's, cancer and HIV. Early attempts date back to the 1960s but the first approved human gene therapy trial was in 1990 for a genetic immune deficiency. Since then, gene therapy has been used successfully to treat various diseases while also facing some setbacks. It delivers therapeutic genes into patient cells using either viral or non-viral vectors in order to potentially offer permanent effects for inherited conditions.
Lgis gene therapy
Gene therapy involves introducing genes into cells to treat disease. The first approved gene therapy treated a girl named Ashanti DeSilva for ADA-SCID in 1990. There are two main types: somatic cell gene therapy targets body cells and is not inherited, while germline gene therapy targets germ cells and is inherited. Gene therapy holds promise for treating genetic diseases but also faces challenges in achieving long-term effects, immune responses, and ethical concerns.
NUCLEIC ACID BASED THERAPEUTIC DELIVERY SYSTEM by pramesh..pptx
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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.
Nucleic Acid Delivery System
Definition, Gene therapy, types of gene therapy, germline gene therapy, somatic cell gene therapy, basic process of gene therapy and potential targets for gene therapy.
Gene therapy
Novel approach to treat, cure or ultimately prevent a disease by changing the expression of a person’s genes
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Gene based and cell based therapy in clinical medicine converted
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NUCLEIC ACID BASED THERAPEUTIC DELIVERY SYSTEM by pramesh..pptx
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gene therapy
- 1. Dr. APJ ABDUL KALAM TECHNICAL UNIVERSITY (Formerly Uttar Pradesh Technical University) LUCKNOW Noida Institute of Engineering & Technology (Pharmacy Institute) Project Title : Gene Therapy Submitted To: Dr. Saumya Das Associate Professor Noida Institute of Engineering & Technology(Pharmacy Institute) Greater Noida Submitted By: Shilajit Das M. Pharm(Pharmacology) Noida Institute of Engineering &Technology (Pharmacy Institute) Greater Noida
- 2. Gene Therapy - Introduction ▪ Gene therapy is the introduction of genes into existing cells to prevent or cure a wide range of diseases. ▪ It is a technique for correcting defective genes responsible for disease development. ▪ The first approved gene therapy experiment occurred on September 14, 1990 in US, when Ashanti DeSilva was treated for ADA-SCID.
- 3. IN VIVO GENE THERAPY ▪ Direct delivery of therapeutic gene into target cell into patients body. ▪ Carried out by viral or non viral vector systems. ▪ It can be the only possible option in patients where individual cells cannot be cultured in vitro in sufficient numbers (e.g. brain cells). ▪ In vivo gene transfer is necessary when cultured cells cannot be re-implanted in patients effectively. Ref : shorturl.at/tzUXY
- 4. EX VIVO GENE THERAPY :- Transplant the modified cells to the patient. Select genetically corrected cells and grow. Introduce the therapeutic genes. Grow the cells in culture Isolate cells with genetic defect from a patient
- 5. Other types of gene therapy GENE AUGMENTATION THERAPY ▪ Most common form of gene therapy. ▪ Foreign gene replaces missing or defective gene. ▪ Eg. Replacement of defective p53 gene by a normal one in liver cancer. GENE INHIBITION THERAPY ▪ Done to block the overproduction of some proteins.- 2 types - Antigene and antisense therapy. - Antigene-blocks transcription using antigene oligonucleotide. - Antisense-blocks transalation using antisense oligonucleotide.
- 6. VECTORS IN GENE THERAPY ▪ To transfer the desired gene into a target cell, a carrier is required. Such vehicles of gene delivery are known as vectors. 2 main classes - Viral vectors. - Non viral vectors Ref : shorturl.at/fvSZ0
- 7. VIRAL VECTORS IN GENE THERAPY ▪ ADENO VIRUS VECTOR SYSTEM ▪ Adeno virus with at DNA genome - good vectors. ▪ Target- non dividing human cell. ▪ Eg. Common cold adenovirus.
- 8. NON VIRAL VECTOR SYSTEM PURE DNA CONSTRUCT ▪ Direct introduction of pure DNA construct into target tissue. ▪ Efficiency of DNA uptake by cells and expression rather low. ▪ Consequently, large quantities of DNA have to be injected periodically. LIPOPLEXES ▪ Lipid DNA complexes: DNA construct surrounded by artificial lipid layer. ▪ Most of it gets degraded by lysosomes..
- 9. Factors to be considered in Gene therapy ▪ How to deliver genes to specific cells, tissue and whole animals? (methods of delivery) ▪ How much and how long the introduced gene will be expressed? ▪ The site and dose of gene delivery ▪ Is there any adverse immunological consequence of both delivery vehicle (Virus) and the gene in animals? ▪ Is there any toxic effects?
- 10. Uses of gene therapy ▪ Clinical gene transfer applications ▪ Vaccine development ▪ Production of transgenic animals ▪ Treatment of cancer and AIDS ▪ Gene discovery ▪ Gene therapy ▪ Enhancing the resistance of plant ▪ Genetically modified organism
- 11. Application of gene therapy ▪ Genetic disorder (deficiency): OTC ▪ Cancer - Genetic predisposition - Mutation in oncogene or tumor suppressor gene ▪ Autoimmunity diseases: rheumatoid arthritis - Delivery of counteracting gene ▪ Diseases involve several genes and the environmental interact: diabetes
- 12. CONCLUSION ▪ Theoretically, gene therapy is the permanent solution for genetic diseases. ▪ But it has several complexities. At its current stage, it is not accessible to most people due to its huge cost. ▪ A breakthrough may come anytime and a day may come when almost every disease will have a gene therapy. ▪ Gene therapy have the potential to revolutionize the practice of medicine.
- 13. REFERENCES ▪ Dubey R.C, A textbook of biotechnology, 1st edition (2004), S Chand and company, New Delhi ▪ Gupta P.K, Elements of Biotechnology, 1stedition (2001), Rastogi Publications, Meerut. ▪ Satyanarayana U, Biotechnology, 1st edition, Book andallied (P) Ltd, Kolkata ▪ http://www.medindia.net/articles/genetherapy_treat ment.htm ▪ http://en.wikipedia.org/wiki/Gene_therapy ▪ shorturl.at/tzUXY ▪ shorturl.at/fvSZ0