Gene therapy
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This document discusses gene therapy, which is an experimental technique that uses genes to treat or prevent disease. It does this in one of three ways: inactivating a mutated gene, introducing a new gene to help fight disease, or replacing a mutated gene with a healthy copy. There are two main types of gene therapy - somatic gene therapy, which targets the body's cells, and germ line gene therapy, which targets egg and sperm cells. Gene delivery can be done ex vivo, by modifying cells outside the body before transplanting them back in, or in vivo by directly modifying cells in the body. Vectors are used to deliver genes into target cells, with the main types being viral vectors and non-viral vectors.
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Gene therapy
Gene therapy is emerging branch of healthcare, we can see that with the possible development it has potential to treat multiple genetic as well as other conditions and disease
hope young scholar can find this presentation useful and i am open to any suggestions
Gene therapy
Gene therapy involves delivering genes to correct genetic defects. It can augment or inhibit gene expression. Key steps include identifying the defective gene, cloning the normal gene, selecting a target cell/tissue, and inserting the gene. Genes can be delivered ex vivo by modifying cells outside the body or in vivo by direct gene transfer. Viral vectors like retroviruses are commonly used, but pose risks like activating proto-oncogenes. Gene therapy aims to treat genetic disorders and acquired diseases.
Gene therapy
This document discusses gene therapy, including:
- Gene therapy involves inserting a normal gene to replace an abnormal gene responsible for disease.
- There are two main types: germ line therapy affects future generations, while somatic therapy only affects the individual.
- The basic process involves researching the defective gene, copying the normal gene, and inserting it into cells.
- Gene therapy could potentially replace defective genes, deliver genes to treat cancer or provide immunity, and stimulate tissue growth or healing. However, it also faces challenges like high costs and ensuring genes only target intended cells. Examples where it has been used include hemophilia, blindness, cancer, and Parkinson's disease.
Gene therapy
This document provides an overview of gene therapy. It discusses the definition of gene therapy as inserting genes into cells to treat disease. The history of gene therapy is described, including early research in the 1970s and the first human clinical trial in 1990 to treat ADA deficiency. The document outlines the main types of gene therapy as somatic, germline, and preventative. It also discusses gene delivery methods using viral and non-viral vectors and the advantages and disadvantages of gene therapy.
Gene therapy in clinical applications
Gene therapy.
Adenovirus
Retrovirus
In vivo and Ex vivo methods
Molecular techniques
Alternative vectors
Biolistic missile
Homologous recombination
Gene therapy
Gene therapy involves inserting functioning genes into patients' cells to treat genetic diseases. It has been studied as a potential treatment for cancers, blood diseases, immune and neurological disorders. There are two main types - somatic cell gene therapy targets specific cells/tissues, while germline gene therapy affects heritable genetic material. Recent progress includes the first approved gene therapy in 1990 and advancements in viral vectors. Major milestones in the 2000s-2010s included treatments for leukemia, ADA-SCID, and retinal diseases. Future prospects are promising with CRISPR/Cas9 offering more precise gene editing and many ongoing clinical trials, though challenges remain around costs, technical expertise, and ethical concerns.
Gene therapy 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.
Challenges faced in gene therapy
Gene therapy involves introducing normal genes into cells to replace missing or defective genes and correct genetic disorders. There are two main types of gene therapy: somatic cell therapy and germline therapy. The document discusses four main challenges of gene therapy: 1) delivering genes to the right tissues and cells and ensuring gene activation, 2) immune responses against foreign genes, 3) disrupting important genes in target cells, and 4) the commercial viability of treating rare genetic disorders given the high costs of clinical trials.
Recommended
Gene therapy
Gene therapy is emerging branch of healthcare, we can see that with the possible development it has potential to treat multiple genetic as well as other conditions and disease
hope young scholar can find this presentation useful and i am open to any suggestions
Gene therapy
Gene therapy involves delivering genes to correct genetic defects. It can augment or inhibit gene expression. Key steps include identifying the defective gene, cloning the normal gene, selecting a target cell/tissue, and inserting the gene. Genes can be delivered ex vivo by modifying cells outside the body or in vivo by direct gene transfer. Viral vectors like retroviruses are commonly used, but pose risks like activating proto-oncogenes. Gene therapy aims to treat genetic disorders and acquired diseases.
Gene therapy
This document discusses gene therapy, including:
- Gene therapy involves inserting a normal gene to replace an abnormal gene responsible for disease.
- There are two main types: germ line therapy affects future generations, while somatic therapy only affects the individual.
- The basic process involves researching the defective gene, copying the normal gene, and inserting it into cells.
- Gene therapy could potentially replace defective genes, deliver genes to treat cancer or provide immunity, and stimulate tissue growth or healing. However, it also faces challenges like high costs and ensuring genes only target intended cells. Examples where it has been used include hemophilia, blindness, cancer, and Parkinson's disease.
Gene therapy
This document provides an overview of gene therapy. It discusses the definition of gene therapy as inserting genes into cells to treat disease. The history of gene therapy is described, including early research in the 1970s and the first human clinical trial in 1990 to treat ADA deficiency. The document outlines the main types of gene therapy as somatic, germline, and preventative. It also discusses gene delivery methods using viral and non-viral vectors and the advantages and disadvantages of gene therapy.
Gene therapy in clinical applications
Gene therapy.
Adenovirus
Retrovirus
In vivo and Ex vivo methods
Molecular techniques
Alternative vectors
Biolistic missile
Homologous recombination
Gene therapy
Gene therapy involves inserting functioning genes into patients' cells to treat genetic diseases. It has been studied as a potential treatment for cancers, blood diseases, immune and neurological disorders. There are two main types - somatic cell gene therapy targets specific cells/tissues, while germline gene therapy affects heritable genetic material. Recent progress includes the first approved gene therapy in 1990 and advancements in viral vectors. Major milestones in the 2000s-2010s included treatments for leukemia, ADA-SCID, and retinal diseases. Future prospects are promising with CRISPR/Cas9 offering more precise gene editing and many ongoing clinical trials, though challenges remain around costs, technical expertise, and ethical concerns.
Gene therapy 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.
Challenges faced in gene therapy
Gene therapy involves introducing normal genes into cells to replace missing or defective genes and correct genetic disorders. There are two main types of gene therapy: somatic cell therapy and germline therapy. The document discusses four main challenges of gene therapy: 1) delivering genes to the right tissues and cells and ensuring gene activation, 2) immune responses against foreign genes, 3) disrupting important genes in target cells, and 4) the commercial viability of treating rare genetic disorders given the high costs of clinical trials.
Strategies of gene therapy
Gene therapy involves introducing DNA into a patient's cells to treat a disease. There are four main strategies of gene therapy: gene augmentation therapy which adds a functional gene; targeted killing of specific cells like cancer cells; targeted mutation correction; and targeted inhibition of gene expression. Gene therapy shows promise for diseases like cystic fibrosis, hemophilia, cancer and more. Challenges remain around safely delivering genes to the right cells and avoiding immune responses.
Gene therapy
Gene therapy involves introducing normal genes into cells to replace missing or defective genes and correct genetic disorders. The first gene therapy treatment occurred in 1990 for SCID. There are two main types - germline therapy would alter the germ cells and be heritable, while somatic therapy only affects the individual. Delivery of genes requires vectors like viruses that can transport the new gene into target cells. Challenges include developing vectors that don't cause immune responses, achieving long-term expression of the gene, and addressing disorders influenced by multiple genes. Risks include insertional mutagenesis and the development of tumors.
Gene therapy: short introduction
Gene therapy involves introducing normal genes into cells containing defective genes to correct genetic disorders. There are four main approaches, including inserting a normal gene to compensate for a nonfunctional one. A viral vector delivers the therapeutic gene into a target cell, where it is inserted into the genome. Different delivery systems include using viruses like retroviruses, adenoviruses, adeno-associated viruses, and herpes simplex viruses. While gene therapy has been used to treat diseases like SCID, Parkinson's, and sickle cell disease, it faces limitations such as immune responses, risk of cancer from uncontrolled insertion, and high expenses.
Gene therapy
Gene therapy involves delivering therapeutic DNA into a patient's cells to treat disease. It was first conceptualized in 1972 and the first FDA-approved gene therapy for ADA-SCID occurred in 1990. Gene therapy targets diseases without other treatments, such as immune disorders, cystic fibrosis, and hemophilia. There are two main types - somatic gene therapy modifies body cells while germline modifies sperm or eggs. Viral and non-viral vectors are used to deliver genes in vivo or ex vivo gene therapy methods.
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.
Gene therapy
Gene therapy aims to treat cystic fibrosis, a genetic disorder caused by mutations in the CFTR gene. Early clinical trials involved delivering a healthy copy of the CFTR gene to the lungs via viral vectors like adenovirus or adeno-associated virus, or non-viral liposomes. While these methods showed some promise, challenges remained in achieving adequate delivery, expression of the gene, and avoiding immune responses. Ongoing research focuses on improving gene transfer methods to make gene therapy more effective for cystic fibrosis.
Applications of gene therapy
Applications of gene therapy and potential disease targeted by gene therapy ( Basic Information about them)
Gene Therapy
Gene therapy is an experimental treatment that involves introducing genetic material into a person’s cells to fight or prevent disease. Researchers are studying gene therapy for a number of diseases, such as severe combined immuno-deficiencies, hemophilia, Parkinson's disease, cancer and even HIV, through a number of different approaches (see video: 'Gene Therapy a new tool to cure human diseases'). A gene can be delivered to a cell using a carrier known as a “vector.” The most common types of vectors used in gene therapy are viruses. The viruses used in gene therapy are altered to make them safe, although some risks still exist with gene therapy. The technology is still in its infancy, but it has been used with some success.
Gene therapy 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
Gene therapy is an experimental technique that involves inserting a normal gene to replace a defective gene responsible for disease. It can replace a mutated disease-causing gene with a healthy copy or inactivate a mutated gene functioning improperly. Common diseases targeted include severe combined immunodeficiency, hemophilia, cystic fibrosis, hemoglobinopathies like thalassemia and sickle cell anemia, Parkinson's disease, and cancer. The first approved gene therapy case occurred in 1990 treating a girl with immune deficiency. Since then, gene therapy has been used to treat metastatic melanoma. Gene therapy can affect the germline permanently or somatic cells temporarily.
Gene therapy
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.
Gene therapy
this presentation about. ex- vivo and in-vivo gene therapy with ex. of cystic fibrosis and SCID disease.
Gene therapy
Gene therapy is the process of inserting genes into cells to prevent, treat or cure wide range of diseases. Gene therapy primarily involves genetic manipulations in animals or humans to correct a disease. Gene augmentation therapy: a DNA is inserted into the Genome to replace the missing gene product.Gene inhibition therapy: the antisense gene inhibits the expression of the dominant gene.
Gene Therapy
Gene therapy involves introducing new genes into cells to treat disease. Researchers are testing approaches like replacing mutated genes, inactivating genes, or introducing new therapeutic genes. Genes can be transferred into somatic or germ cells using vectors like adeno-associated viral or lentiviral vectors. Examples discussed include successful gene therapy trials for sickle cell disease and hemophilia that showed therapeutic levels of proteins and correction of disease symptoms for over a year. However, challenges remain regarding safe and targeted delivery of genes, immune responses, and ethical issues surrounding gene editing and enhancement.
Gene therapy : Basic introduction
This document provides an introduction to gene therapy. It defines gene therapy as a technique for correcting defective genes responsible for disease development by introducing one or more foreign genes into an organism. The document outlines the basic components of DNA, genes, and chromosomes. It then describes the different types and strategies of gene therapy, including replacement, knockout, suicide, and immunomodulatory gene therapy. The approaches, advantages, and problems of gene therapy are summarized.
Basics of Gene Therapy
Gene is the basic physical unit of inheritance that passed information from parents to offspring. Genes are arranged, one after another, on structure called chromosome.
A gene is region of DNA that encodes function and chromosome consist of long DNA strands containing many genes.
A human chromosome can have up to 500 million base pair of DNA with thousands of genes.
Technique responsible for correcting the defective genes responsible for disease development is called Gene Therapy
Gene therapy
Gene therapy involves introducing genetic material into host cells to treat diseases caused by genetic mutations. It has been used to treat several conditions like ADA-SCID, cystic fibrosis, and inherited retinal diseases. Various gene delivery systems exist including viral vectors like adenovirus, AAV, and retroviruses. Strategies for gene therapy include gene augmentation, inhibition, targeting, assisted killing, and prodrug therapy. While promising, gene therapy still faces challenges like improving delivery methods and reducing immune responses.
Gene therapy ppt for students and staff
Surviving animals produced by nuclear transfer are healthy.
There, is a substantial loss of individual before and after birth some of the cloned animals display abnormalities.
Abnormlities such as increased birth weight.
Dna methylation and histone modification of the original donor cell is inappropriate maintained in the cells of the recipient animals.
INVIVO:
The direct delivery of the therapeutic gene into the target cells of a particular tissue.
Genes are changed in cells when the cells are still in the body.
These include liver, muscle, skin, spleen, lung, brain and blood cells.
Carried out by viral and non viral vector system
EXVIVO:
Cells are modified outside the body and then transplanted back in again.
SCID is a inherited immune disorder associated with T-lymphocyte and B-lymphocytes dysfunction.
Gene encodes for adenosine deaminase.
The patients of SCID(lacking ADA) suffer from infectious diseases and die at an young age.
Gene therapy
This document summarizes the history and development of gene therapy from the 1950s to the present. It discusses key events like the isolation of genes in the 1970s and 1980s and the first human gene therapy treatment in 1990. It outlines various gene therapy strategies like ex vivo and in vivo approaches and delivery methods like viruses, liposomes, microinjection, and electroporation. Challenges of gene therapy are also summarized like ethical issues, high costs, and ensuring safety. The document aims to provide an overview of the progress of gene therapy and remaining barriers to its clinical application and acceptance.
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
GENE THERAPY
S.RAM PRAKASH
IIIrdBSC BIOTECHNOLOGY, AYYA NADAR JANAKI AMMA COLLEGE, SIVAKASI, TAMIL NADU.
Gene therapy
This document discusses gene therapy, including its introduction, types, strategies, approaches, methods, target cells, and vectors. Gene therapy involves introducing genes into cells to treat genetic defects or diseases. There are two main types - somatic gene therapy targets somatic cells to treat an individual without inheritance, while germline gene therapy targets germ cells to make the therapy heritable. Common strategies include gene augmentation, targeted killing of diseased cells, inhibition of gene expression, and correction of mutated genes. Methods include ex vivo therapy of cultured cells outside the body and in vivo therapy of direct gene transfer inside the body. Viral and non-viral vectors are used to deliver therapeutic genes to different target cells depending on the disease.
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Strategies of gene therapy
Gene therapy involves introducing DNA into a patient's cells to treat a disease. There are four main strategies of gene therapy: gene augmentation therapy which adds a functional gene; targeted killing of specific cells like cancer cells; targeted mutation correction; and targeted inhibition of gene expression. Gene therapy shows promise for diseases like cystic fibrosis, hemophilia, cancer and more. Challenges remain around safely delivering genes to the right cells and avoiding immune responses.
Gene therapy
Gene therapy involves introducing normal genes into cells to replace missing or defective genes and correct genetic disorders. The first gene therapy treatment occurred in 1990 for SCID. There are two main types - germline therapy would alter the germ cells and be heritable, while somatic therapy only affects the individual. Delivery of genes requires vectors like viruses that can transport the new gene into target cells. Challenges include developing vectors that don't cause immune responses, achieving long-term expression of the gene, and addressing disorders influenced by multiple genes. Risks include insertional mutagenesis and the development of tumors.
Gene therapy: short introduction
Gene therapy involves introducing normal genes into cells containing defective genes to correct genetic disorders. There are four main approaches, including inserting a normal gene to compensate for a nonfunctional one. A viral vector delivers the therapeutic gene into a target cell, where it is inserted into the genome. Different delivery systems include using viruses like retroviruses, adenoviruses, adeno-associated viruses, and herpes simplex viruses. While gene therapy has been used to treat diseases like SCID, Parkinson's, and sickle cell disease, it faces limitations such as immune responses, risk of cancer from uncontrolled insertion, and high expenses.
Gene therapy
Gene therapy involves delivering therapeutic DNA into a patient's cells to treat disease. It was first conceptualized in 1972 and the first FDA-approved gene therapy for ADA-SCID occurred in 1990. Gene therapy targets diseases without other treatments, such as immune disorders, cystic fibrosis, and hemophilia. There are two main types - somatic gene therapy modifies body cells while germline modifies sperm or eggs. Viral and non-viral vectors are used to deliver genes in vivo or ex vivo gene therapy methods.
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.
Gene therapy
Gene therapy aims to treat cystic fibrosis, a genetic disorder caused by mutations in the CFTR gene. Early clinical trials involved delivering a healthy copy of the CFTR gene to the lungs via viral vectors like adenovirus or adeno-associated virus, or non-viral liposomes. While these methods showed some promise, challenges remained in achieving adequate delivery, expression of the gene, and avoiding immune responses. Ongoing research focuses on improving gene transfer methods to make gene therapy more effective for cystic fibrosis.
Applications of gene therapy
Applications of gene therapy and potential disease targeted by gene therapy ( Basic Information about them)
Gene Therapy
Gene therapy is an experimental treatment that involves introducing genetic material into a person’s cells to fight or prevent disease. Researchers are studying gene therapy for a number of diseases, such as severe combined immuno-deficiencies, hemophilia, Parkinson's disease, cancer and even HIV, through a number of different approaches (see video: 'Gene Therapy a new tool to cure human diseases'). A gene can be delivered to a cell using a carrier known as a “vector.” The most common types of vectors used in gene therapy are viruses. The viruses used in gene therapy are altered to make them safe, although some risks still exist with gene therapy. The technology is still in its infancy, but it has been used with some success.
Gene therapy 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
Gene therapy is an experimental technique that involves inserting a normal gene to replace a defective gene responsible for disease. It can replace a mutated disease-causing gene with a healthy copy or inactivate a mutated gene functioning improperly. Common diseases targeted include severe combined immunodeficiency, hemophilia, cystic fibrosis, hemoglobinopathies like thalassemia and sickle cell anemia, Parkinson's disease, and cancer. The first approved gene therapy case occurred in 1990 treating a girl with immune deficiency. Since then, gene therapy has been used to treat metastatic melanoma. Gene therapy can affect the germline permanently or somatic cells temporarily.
Gene therapy
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.
Gene therapy
this presentation about. ex- vivo and in-vivo gene therapy with ex. of cystic fibrosis and SCID disease.
Gene therapy
Gene therapy is the process of inserting genes into cells to prevent, treat or cure wide range of diseases. Gene therapy primarily involves genetic manipulations in animals or humans to correct a disease. Gene augmentation therapy: a DNA is inserted into the Genome to replace the missing gene product.Gene inhibition therapy: the antisense gene inhibits the expression of the dominant gene.
Gene Therapy
Gene therapy involves introducing new genes into cells to treat disease. Researchers are testing approaches like replacing mutated genes, inactivating genes, or introducing new therapeutic genes. Genes can be transferred into somatic or germ cells using vectors like adeno-associated viral or lentiviral vectors. Examples discussed include successful gene therapy trials for sickle cell disease and hemophilia that showed therapeutic levels of proteins and correction of disease symptoms for over a year. However, challenges remain regarding safe and targeted delivery of genes, immune responses, and ethical issues surrounding gene editing and enhancement.
Gene therapy : Basic introduction
This document provides an introduction to gene therapy. It defines gene therapy as a technique for correcting defective genes responsible for disease development by introducing one or more foreign genes into an organism. The document outlines the basic components of DNA, genes, and chromosomes. It then describes the different types and strategies of gene therapy, including replacement, knockout, suicide, and immunomodulatory gene therapy. The approaches, advantages, and problems of gene therapy are summarized.
Basics of Gene Therapy
Gene is the basic physical unit of inheritance that passed information from parents to offspring. Genes are arranged, one after another, on structure called chromosome.
A gene is region of DNA that encodes function and chromosome consist of long DNA strands containing many genes.
A human chromosome can have up to 500 million base pair of DNA with thousands of genes.
Technique responsible for correcting the defective genes responsible for disease development is called Gene Therapy
Gene therapy
Gene therapy involves introducing genetic material into host cells to treat diseases caused by genetic mutations. It has been used to treat several conditions like ADA-SCID, cystic fibrosis, and inherited retinal diseases. Various gene delivery systems exist including viral vectors like adenovirus, AAV, and retroviruses. Strategies for gene therapy include gene augmentation, inhibition, targeting, assisted killing, and prodrug therapy. While promising, gene therapy still faces challenges like improving delivery methods and reducing immune responses.
Gene therapy ppt for students and staff
Surviving animals produced by nuclear transfer are healthy.
There, is a substantial loss of individual before and after birth some of the cloned animals display abnormalities.
Abnormlities such as increased birth weight.
Dna methylation and histone modification of the original donor cell is inappropriate maintained in the cells of the recipient animals.
INVIVO:
The direct delivery of the therapeutic gene into the target cells of a particular tissue.
Genes are changed in cells when the cells are still in the body.
These include liver, muscle, skin, spleen, lung, brain and blood cells.
Carried out by viral and non viral vector system
EXVIVO:
Cells are modified outside the body and then transplanted back in again.
SCID is a inherited immune disorder associated with T-lymphocyte and B-lymphocytes dysfunction.
Gene encodes for adenosine deaminase.
The patients of SCID(lacking ADA) suffer from infectious diseases and die at an young age.
Gene therapy
This document summarizes the history and development of gene therapy from the 1950s to the present. It discusses key events like the isolation of genes in the 1970s and 1980s and the first human gene therapy treatment in 1990. It outlines various gene therapy strategies like ex vivo and in vivo approaches and delivery methods like viruses, liposomes, microinjection, and electroporation. Challenges of gene therapy are also summarized like ethical issues, high costs, and ensuring safety. The document aims to provide an overview of the progress of gene therapy and remaining barriers to its clinical application and acceptance.
GENE THERAPY.1.pptx
Above slides consist of detailed information of gene therapy,including types, methods, future needs along with advantages and disadvantages
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Gene therapy - current status and future perspective
Gene therapy - current status and future perspective
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Gene therapy
GENE THERAPY
S.RAM PRAKASH
IIIrdBSC BIOTECHNOLOGY, AYYA NADAR JANAKI AMMA COLLEGE, SIVAKASI, TAMIL NADU.
Gene therapy
This document discusses gene therapy, including its introduction, types, strategies, approaches, methods, target cells, and vectors. Gene therapy involves introducing genes into cells to treat genetic defects or diseases. There are two main types - somatic gene therapy targets somatic cells to treat an individual without inheritance, while germline gene therapy targets germ cells to make the therapy heritable. Common strategies include gene augmentation, targeted killing of diseased cells, inhibition of gene expression, and correction of mutated genes. Methods include ex vivo therapy of cultured cells outside the body and in vivo therapy of direct gene transfer inside the body. Viral and non-viral vectors are used to deliver therapeutic genes to different target cells depending on the disease.
Gene Therapy
Gene therapy is an experimental technique that uses genes to treat or prevent disease. There are two types of gene therapy - somatic gene therapy, which treats cells of the body but not reproductive cells, and germline gene therapy, which treats reproductive cells and can be passed to future generations. There are several techniques for gene therapy including gene augmentation therapy, which adds a functional gene to replace a non-functioning one; gene inhibition therapy, which inhibits or interferes with a disease-causing gene; and gene killing therapy, which inserts a "suicide gene" to kill specific diseased cells. A key challenge is safely delivering the new gene to the right cells and ensuring it is expressed without disrupting other genes or triggering
Gene therapy
This document provides an overview of gene therapy including its principles, approaches, development, types, vectors, delivery methods, examples, advantages, and disadvantages. Gene therapy involves inserting genes into cells to treat diseases caused by defective genes. There are two main approaches - germline gene therapy, which alters the germ cells and is passed to offspring, and somatic gene therapy, which alters non-reproductive cells only in the individual. Gene therapy development involves pre-clinical and clinical trials. Vectors like viruses are used to deliver therapeutic genes. Examples include treating severe combined immunodeficiency. While gene therapy has potential benefits, there are also risks like immune responses and ensuring genes reach the right cells.
Gene therapy: How does it work.
Gene therapy is an experimental technique that uses genes to treat or prevent disease which allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery.
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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.
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Gene therapy involves introducing genes into a person's cells to treat disease. There are two main types - somatic gene therapy targets body cells and is not inherited, while germline gene therapy targets sperm or egg cells and is inherited. Gene therapy strategies include gene augmentation to replace a faulty gene, targeted killing of diseased cells, inhibiting gene expression, and correcting defective genes. Methods of delivery include ex vivo gene therapy where cells are modified outside the body and reintroduced, and in vivo gene therapy where genes are directly delivered inside the body. Viral vectors like retroviruses and adenoviruses are commonly used delivery methods. Gene therapy offers cures for genetic diseases and cancer treatments.
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Cancer is one of the most challenging diseases and up until now. One of the most challenging things about cancer treatment is not the cure itself but the differentiation between the tumor cells and the normal cells. Most of the medical treatments of the cancer today cannot differentiate between the cancer cells and the normal one as well as it damages the hall tissue and it is still considered as a low-effect treatment to be applied in cancer. One of the most popular treatments of this kind is chemotherapy which is known for damaging the hall cells, cancer, and normal ones. Our research is focusing on generating a new therapy that can target the cancer cell itself so it will give us more efficiency ratio to stop cancer and will keep the other cells without any damage. We will use an antibody body for the protein antigen ErbB-2 which is located rabidly in the lung cancer cells' membrane surface. These antibodies will be produced by the immune system so it will target the tumor cells especially and stop the cell growth and damage it in some cases.
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Gene therapy
This document discusses gene therapy and its various aspects. It defines gene therapy as a method of treatment using genes or DNA instead of drugs to treat diseases caused by defective genes. The DNA is delivered into cells using vectors like viruses. The document discusses two main types of gene therapy - somatic cell therapy which affects only the treated individual, and germ line therapy which affects future generations. It also discusses various gene therapy strategies, vectors, methods of gene delivery and challenges in gene therapy.
Gene therapy.
This document outlines a seminar topic on somatic cell gene therapy. It discusses the different types of gene therapy, including somatic cell gene therapy and germ line gene therapy. Somatic cell gene therapy involves transferring genes into any cell other than germ cells to treat an individual patient only. The document then describes the various types of somatic cell gene therapy in more detail, such as ex-vivo gene therapy, in-vivo gene therapy using viruses, and antisense gene therapy. It also reviews some advantages and disadvantages of gene therapy.
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This document provides information on various types of cancer treatments including immunotherapy, prodrug therapy, and targeted therapies. It discusses how immunotherapy works to boost the immune system's natural defenses to fight cancer. Examples of immunotherapy approaches include monoclonal antibodies, cancer vaccines, and non-specific immunotherapies like cytokines. Prodrugs are inactive compounds that are metabolized into active drugs inside the body. Targeted therapies work by interfering with specific proteins that help tumors grow and spread.
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This document discusses gene therapy, which involves inserting normal genes into patients to replace abnormal genes that cause diseases. The most common form replaces a mutated gene with a healthy copy. Gene therapy can be used for diseases like severe combined immunodeficiencies, haemophilia, Parkinson's disease, cancer, and cystic fibrosis. There are two main types: germline gene therapy results in permanent changes that are inherited, while somatic gene therapy only affects the patient's cells and is not passed to offspring. The document outlines the process researchers use to design gene therapies.
Germ line gene therapy
Gene therapy have the potential to revolutionize the practice of medicine. A breakthrough may come anytime and a day may come when almost every disease will have a 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.
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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.
Health biotechnology
This document provides an outline for a lecture on health biotechnology. It begins with an introduction to the topic and lists various course contents that will be covered, including molecular basis of disease, genetic markers, detection of infectious agents, immunization techniques, organ transplantation, applications of transgenic animals, drug delivery systems, and uses of stem cell technology. Reference books on medical biotechnology and health and pharmaceutical biotechnology are also provided. The document then provides an outline of the lecture, discussing introduction to health biotechnology, its applications in areas like drug production, pharmacogenomics, and gene therapy. It also discusses genetic testing.
Gene therapy
Gene therapy holds promise for treating diseases like cancer, HIV, and genetic disorders. It works by inserting a normal gene to replace an abnormal one causing disease. Viruses are often used as vectors to deliver the new gene into cells. There are two main types: germline alters genes in reproductive cells and is unethical, while somatic only alters genes in other body cells. Gene therapy has had some success but also deaths, and faces ethical issues regarding cost, safety, and enhancement versus treatment of disease. It remains experimental but could help many if developed responsibly.
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Gene therapy
- 1. Kosar Rahimi December 2017 Sharif University of Technology Department of Chemical and Petroleum Engineering
- 2. What are genes? Units of inheritance Encoding instructions on how to make proteins 1/15
- 3. What is gene therapy? Gene therapy is an experimental technique that uses genes to treat or prevent disease. (instead of using drugs or surgery) Inactivating, or “knocking out,” a mutated gene that is functioning improperly. Introducing a new gene into the body to help fight a disease. Replacing a mutated gene that causes disease with a healthy copy of the gene. 2/15
- 4. Overall view on Replacing 3/15
- 5. Types of gene therapy depend on target Somatic gene therapy • body Germ line gene therapy • Egg and sperm 4/15
- 6. Types of somatic gene therapy Ex vivo Cells are modified out outside the body and then transplanted back in again. In vivo Cells are changed in cells when the cells are still in the body. 5/15
- 7. Ex vivo gene therapy 6/15
- 8. in vivo gene therapy 7/15
- 9. Vectors in gene therapy Vectors are vehicles of gene delivery into target cells. 2 main classes: o Viral vectors o Non viral vectors 8/15
- 11. Why Non viral vectors? • unexpected immune response • oncogenic effects problems occurring with viral vectors Non viral vectors Physical method Chemical method 10/15
- 12. Non viral vectors-physical approaches 11/15 Carrier-free gene delivery Naked DNA delivery
- 13. Non viral vectors-chemical approaches categorization of chemical carrier protecting the DNA target delivery to specific cell types increasing delivery of DNA to the cytosol or nucleus controlled released of DNA 12/15
- 14. Disadvantages of gene therapy Very high cost Having potential to give rise in other disorders Ethical issues 13/15
- 15. Success cases of gene therapy Hereditary blindness Parkinson's disease Immune deficiencies cancer Blood disease Hemophilia 14/15
- 16. 15/15