Challenges faced in gene therapy
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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.
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Gene therapy
Gene therapy involves using genes as pharmaceutical agents to treat disease. There are four main approaches to gene therapy: inserting a normal gene to compensate for a nonfunctional one, suppressing abnormal gene expression, repairing an abnormal gene through selective reverse mutation, or changing gene regulation. Gene therapy aims to cure the underlying causes of diseases, whereas conventional therapies usually just relieve symptoms. Gene therapy also has the potential for permanent or inheritable effects, unlike most conventional therapies. Common methods for delivering genes include viral vectors like retroviruses, adenoviruses, and AAV, as well as non-viral methods like naked DNA, electroporation, and liposomes.
Gene therapy with viral and non viral vectors.pptx
This document discusses gene therapy. It begins by explaining what genes are and how genetic disorders can result from altered genes. Gene therapy is then defined as inserting genes into cells to treat a disease, such as replacing a defective gene with a functional one. Several methods of gene therapy are described, including using viruses or non-viral vectors to deliver therapeutic genes. Common viral vectors like retroviruses, adenoviruses, and adeno-associated viruses are explained in more detail, along with considerations for the ideal vector. The document concludes by focusing specifically on adenoviruses and adeno-associated viruses as two commonly used viral vectors for gene therapy.
Non viral gene transfer
This document discusses non-viral gene transfer methods. It describes various techniques for direct delivery of naked DNA including electroporation, gene guns, sonoporation, magnetofection, hydrodynamic delivery, and microinjections. It also discusses various non-viral vectors for gene delivery including oligonucleotides, liposomes, lipoplexes, polymersomes, polyplexes, dendrimers, inorganic nanoparticles, and cell-penetrating peptides. Each method is described in terms of its mechanism of delivery, advantages, disadvantages and suitable target tissues. The document provides an overview of non-viral gene expression systems and delivery methods.
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
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 delivery system
The document summarizes a gene delivery system project submitted by two students. It describes how gene delivery systems provide a new perspective for modern medicine by allowing therapeutic genes to be inserted into target cells using viral or non-viral vectors. Viral vectors commonly used include adenoviruses, retroviruses, and adeno-associated viruses. Non-viral methods include using naked DNA, physical methods like electroporation, and chemical methods like lipoplexes and dendrimers. The conclusion states that gene delivery systems allow medicines to be directly injected into cells to cure diseases by attacking cells at the genetic level.
Gene therapy and gene delivery systems
Genes carry hereditary information and encode proteins. Gene therapy aims to treat diseases by correcting defective genes through various approaches like inserting a normal gene or repairing an abnormal gene. Early research in the 1980s involved inserting human genes into bacteria. The first human gene therapy treated a girl for SCID in 1990 by inserting a normal gene into her white blood cells. Viral vectors like retroviruses and adenoviruses as well as non-viral methods can deliver genes, but all methods face challenges like short-lived effects, immune responses, and difficulties treating multi-gene disorders and diseases. While progress has been made, setbacks include a death in 1999 from an immune response and some children developing cancer from viral vectors.
Gene therapy
Gene therapy , Nucleic acids , GENES , ADA-SCID(Severe combined immunodeficiency) , APPROACHES IN GENE THERAPY : Types of gene therapy , somatic gene therapy , germ line gene therapy , Gene modification , gene replacement , gene correction , gene augmentation , Gene transfer methods , viral gene transfer (biological) , non viral gene transfer : physical method ,chemical method , retrovirus vector system , adenovirus vector system , Adeno associated virus vector , Herpex simplex virus vector , Electroporation , Microinjection , Gene Gun , Calcium phosphate mediated DNA transfer , Liposome mediated gene transfer , Potential target diseases for Gene Therapy , GENE THERAPY USED TO TREAT TYPE I DIABETES , GENE THERAPY FOR CANCER TREATMENT , PARKINSON’S DISEASE , GENE THERAPY TREATMENT FOR ADA-SCID , GENE THERAPY FOR CYSTIC FIBROSI S, HEMOPHILIA , BLINDNESS
Gene therapy , Nucleic acids , GENES , ADA-SCID(Severe combined immunodeficiency) , APPROACHES IN GENE THERAPY : Types of gene therapy , somatic gene therapy , germ line gene therapy , Gene modification , gene replacement , gene correction , gene augmentation , Gene transfer methods , viral gene transfer (biological) , non viral gene transfer : physical method ,chemical method , retrovirus vector system , adenovirus vector system , Adeno associated virus vector , Herpex simplex virus vector , Electroporation , Microinjection , Gene Gun , Calcium phosphate mediated DNA transfer , Liposome mediated gene transfer , Potential target diseases for Gene Therapy , GENE THERAPY USED TO TREAT TYPE I DIABETES , GENE THERAPY FOR CANCER TREATMENT , PARKINSON’S DISEASE , GENE THERAPY TREATMENT FOR ADA-SCID , GENE THERAPY FOR CYSTIC FIBROSI S, HEMOPHILIA , BLINDNESS
Recommended
Gene therapy
Gene therapy involves using genes as pharmaceutical agents to treat disease. There are four main approaches to gene therapy: inserting a normal gene to compensate for a nonfunctional one, suppressing abnormal gene expression, repairing an abnormal gene through selective reverse mutation, or changing gene regulation. Gene therapy aims to cure the underlying causes of diseases, whereas conventional therapies usually just relieve symptoms. Gene therapy also has the potential for permanent or inheritable effects, unlike most conventional therapies. Common methods for delivering genes include viral vectors like retroviruses, adenoviruses, and AAV, as well as non-viral methods like naked DNA, electroporation, and liposomes.
Gene therapy with viral and non viral vectors.pptx
This document discusses gene therapy. It begins by explaining what genes are and how genetic disorders can result from altered genes. Gene therapy is then defined as inserting genes into cells to treat a disease, such as replacing a defective gene with a functional one. Several methods of gene therapy are described, including using viruses or non-viral vectors to deliver therapeutic genes. Common viral vectors like retroviruses, adenoviruses, and adeno-associated viruses are explained in more detail, along with considerations for the ideal vector. The document concludes by focusing specifically on adenoviruses and adeno-associated viruses as two commonly used viral vectors for gene therapy.
Non viral gene transfer
This document discusses non-viral gene transfer methods. It describes various techniques for direct delivery of naked DNA including electroporation, gene guns, sonoporation, magnetofection, hydrodynamic delivery, and microinjections. It also discusses various non-viral vectors for gene delivery including oligonucleotides, liposomes, lipoplexes, polymersomes, polyplexes, dendrimers, inorganic nanoparticles, and cell-penetrating peptides. Each method is described in terms of its mechanism of delivery, advantages, disadvantages and suitable target tissues. The document provides an overview of non-viral gene expression systems and delivery methods.
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
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 delivery system
The document summarizes a gene delivery system project submitted by two students. It describes how gene delivery systems provide a new perspective for modern medicine by allowing therapeutic genes to be inserted into target cells using viral or non-viral vectors. Viral vectors commonly used include adenoviruses, retroviruses, and adeno-associated viruses. Non-viral methods include using naked DNA, physical methods like electroporation, and chemical methods like lipoplexes and dendrimers. The conclusion states that gene delivery systems allow medicines to be directly injected into cells to cure diseases by attacking cells at the genetic level.
Gene therapy and gene delivery systems
Genes carry hereditary information and encode proteins. Gene therapy aims to treat diseases by correcting defective genes through various approaches like inserting a normal gene or repairing an abnormal gene. Early research in the 1980s involved inserting human genes into bacteria. The first human gene therapy treated a girl for SCID in 1990 by inserting a normal gene into her white blood cells. Viral vectors like retroviruses and adenoviruses as well as non-viral methods can deliver genes, but all methods face challenges like short-lived effects, immune responses, and difficulties treating multi-gene disorders and diseases. While progress has been made, setbacks include a death in 1999 from an immune response and some children developing cancer from viral vectors.
Gene therapy
Gene therapy , Nucleic acids , GENES , ADA-SCID(Severe combined immunodeficiency) , APPROACHES IN GENE THERAPY : Types of gene therapy , somatic gene therapy , germ line gene therapy , Gene modification , gene replacement , gene correction , gene augmentation , Gene transfer methods , viral gene transfer (biological) , non viral gene transfer : physical method ,chemical method , retrovirus vector system , adenovirus vector system , Adeno associated virus vector , Herpex simplex virus vector , Electroporation , Microinjection , Gene Gun , Calcium phosphate mediated DNA transfer , Liposome mediated gene transfer , Potential target diseases for Gene Therapy , GENE THERAPY USED TO TREAT TYPE I DIABETES , GENE THERAPY FOR CANCER TREATMENT , PARKINSON’S DISEASE , GENE THERAPY TREATMENT FOR ADA-SCID , GENE THERAPY FOR CYSTIC FIBROSI S, HEMOPHILIA , BLINDNESS
Gene therapy , Nucleic acids , GENES , ADA-SCID(Severe combined immunodeficiency) , APPROACHES IN GENE THERAPY : Types of gene therapy , somatic gene therapy , germ line gene therapy , Gene modification , gene replacement , gene correction , gene augmentation , Gene transfer methods , viral gene transfer (biological) , non viral gene transfer : physical method ,chemical method , retrovirus vector system , adenovirus vector system , Adeno associated virus vector , Herpex simplex virus vector , Electroporation , Microinjection , Gene Gun , Calcium phosphate mediated DNA transfer , Liposome mediated gene transfer , Potential target diseases for Gene Therapy , GENE THERAPY USED TO TREAT TYPE I DIABETES , GENE THERAPY FOR CANCER TREATMENT , PARKINSON’S DISEASE , GENE THERAPY TREATMENT FOR ADA-SCID , GENE THERAPY FOR CYSTIC FIBROSI S, HEMOPHILIA , BLINDNESS
Gene therapy
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.
Gene therapy ex vivo method
This document discusses ex vivo gene therapy methods. It involves 3 main steps:
1) Isolation of cells from a patient with a genetic defect
2) Culturing and genetically modifying the cells to correct the defect
3) Transplanting the modified cells back into the patient. As the patient's own cells are used, there is no risk of immune rejection. Viral and non-viral vectors are used to deliver therapeutic genes to the cultured cells. Ex vivo gene therapy is applicable to tissues that can be removed, modified and reintroduced into the body.
Cell culture techniques
Equipments used , types of culture and media, subculturing, secondary culture, finite & continuous cell lines, cryopreservation and applications of cell culture
Cellular & Gene Therapy
These slides discusses on cellular and gene therapy: the use of cells and genes to treat disease. These therapies can be effective on a wide range of previously untreated diseases, such as hematological, ocular, neurodegenerative diseases, and several types of cancers.
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.
13-3 cell transformation
This document discusses cell transformation, which is a change to a cell's DNA. It describes three methods of transforming cells:
1) Transforming bacteria cells using plasmids containing foreign DNA that can be inserted into the plasmid. The recombinant plasmids can then deliver the DNA into the bacterial cell.
2) Transforming plant cells either by using the bacterium Agrobacterium tumefaciens to transfer recombinant plasmids, or by using a gene gun to inject DNA-coated particles into plant cells.
3) Transforming animal cells by injecting foreign DNA directly into egg nuclei, or using gene replacement to "knock out" an existing gene.
Gene therapy
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.
Transfection
The document discusses various methods of transfection in animals. Transfection is the process of introducing nucleic acids into eukaryotic cells. It describes viral transfection using bacteria like Agrobacterium tumefaciens and viruses. Non-viral methods include chemical transfection using calcium phosphate, liposomes, polyamines. Mechanical transfection employs microinjection or particle bombardment. Common chemical methods are calcium phosphate precipitation, polyplexes, and liposomes/lipoplexes. Viruses used are retroviruses, adenoviruses, adeno-associated viruses. Bacterial and viral vectors allow for integration into the host genome while chemical and mechanical are often transient.
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This document discusses 3D cell culture techniques. It defines 3D cell culture as permitting biological cells to grow and interact in all three dimensions, mimicking their natural environment. This is an improvement over 2D cultures where cells grow unnaturally. The document describes various 3D culture methods including scaffold-based techniques using polymeric scaffolds or biological scaffolds, and non-scaffold methods like hanging drop plates, spheroid plates, microfluidics, and gels. It also discusses bioreactors and lists applications of 3D cultures.
Gene therapy in clinical applications
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Gene expression is the process by which genetic information encoded in DNA is converted into structures and functions within cells. It involves three main steps: replication, transcription, and translation. Replication copies DNA to produce identical daughter molecules. Transcription converts the DNA sequence into messenger RNA (mRNA). Translation then uses the mRNA to produce proteins through the joining of amino acids specified by the mRNA's codon sequence. These three steps together allow genetic information to direct the production of the RNA and protein molecules that drive cellular functions and inheritance of traits.
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This document discusses gene therapy approaches including ex-vivo and in-vivo gene therapy. Ex-vivo gene therapy involves removing cells from the body, introducing therapeutic genes in culture, and reintroducing the modified cells. In-vivo gene therapy directly delivers therapeutic genes into target cells using viral or non-viral vectors. Examples of each approach are provided, such as using ex-vivo gene therapy to treat adenosine deaminase deficiency and in-vivo gene therapy to treat cystic fibrosis. In conclusion, while gene therapy has potential, it faces complexity and high costs, limiting current accessibility.
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Recombinant viral vectors are genetic engineering tools commonly used for gene transfer purpose with high transfection efficiency and site specific gene insertion.
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Gene therapy is the modern techniques of treatment of various diseases and disorders.
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.
Inactivating, or “knocking out,” a mutated gene that is functioning improperly.
The first approved gene therapy experiment occurred on September1990 in US, when Ashanti DeSilva was treated for ADA-SCID.
Under the direction of William French Anderson, at the National Institutes of Health (NIH),
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Genomics and proteomics in drug discovery and development
This document discusses the role of genomics and proteomics in drug discovery and development. It explains that genomics and proteomics technologies can help identify new drug targets by comparing gene and protein expression between healthy and diseased cells. Proteomics in particular analyzes changes in protein levels and can quantify individual proteins using techniques like 2D gel electrophoresis and mass spectrometry. The integration of genomics and proteomics provides a more comprehensive understanding of biological systems and is improving the drug discovery process.
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Gene therapy
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.
Gene therapy ex vivo method
This document discusses ex vivo gene therapy methods. It involves 3 main steps:
1) Isolation of cells from a patient with a genetic defect
2) Culturing and genetically modifying the cells to correct the defect
3) Transplanting the modified cells back into the patient. As the patient's own cells are used, there is no risk of immune rejection. Viral and non-viral vectors are used to deliver therapeutic genes to the cultured cells. Ex vivo gene therapy is applicable to tissues that can be removed, modified and reintroduced into the body.
Cell culture techniques
Equipments used , types of culture and media, subculturing, secondary culture, finite & continuous cell lines, cryopreservation and applications of cell culture
Cellular & Gene Therapy
These slides discusses on cellular and gene therapy: the use of cells and genes to treat disease. These therapies can be effective on a wide range of previously untreated diseases, such as hematological, ocular, neurodegenerative diseases, and several types of cancers.
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.
13-3 cell transformation
This document discusses cell transformation, which is a change to a cell's DNA. It describes three methods of transforming cells:
1) Transforming bacteria cells using plasmids containing foreign DNA that can be inserted into the plasmid. The recombinant plasmids can then deliver the DNA into the bacterial cell.
2) Transforming plant cells either by using the bacterium Agrobacterium tumefaciens to transfer recombinant plasmids, or by using a gene gun to inject DNA-coated particles into plant cells.
3) Transforming animal cells by injecting foreign DNA directly into egg nuclei, or using gene replacement to "knock out" an existing gene.
Gene therapy
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.
Transfection
The document discusses various methods of transfection in animals. Transfection is the process of introducing nucleic acids into eukaryotic cells. It describes viral transfection using bacteria like Agrobacterium tumefaciens and viruses. Non-viral methods include chemical transfection using calcium phosphate, liposomes, polyamines. Mechanical transfection employs microinjection or particle bombardment. Common chemical methods are calcium phosphate precipitation, polyplexes, and liposomes/lipoplexes. Viruses used are retroviruses, adenoviruses, adeno-associated viruses. Bacterial and viral vectors allow for integration into the host genome while chemical and mechanical are often transient.
3D cell cultures
This document discusses 3D cell culture techniques. It defines 3D cell culture as permitting biological cells to grow and interact in all three dimensions, mimicking their natural environment. This is an improvement over 2D cultures where cells grow unnaturally. The document describes various 3D culture methods including scaffold-based techniques using polymeric scaffolds or biological scaffolds, and non-scaffold methods like hanging drop plates, spheroid plates, microfluidics, and gels. It also discusses bioreactors and lists applications of 3D cultures.
Gene therapy in clinical applications
Gene therapy.
Adenovirus
Retrovirus
In vivo and Ex vivo methods
Molecular techniques
Alternative vectors
Biolistic missile
Homologous recombination
Gene expression
Gene expression is the process by which genetic information encoded in DNA is converted into structures and functions within cells. It involves three main steps: replication, transcription, and translation. Replication copies DNA to produce identical daughter molecules. Transcription converts the DNA sequence into messenger RNA (mRNA). Translation then uses the mRNA to produce proteins through the joining of amino acids specified by the mRNA's codon sequence. These three steps together allow genetic information to direct the production of the RNA and protein molecules that drive cellular functions and inheritance of traits.
antisense technology
Antisense technology uses short DNA sequences called oligonucleotides that are complementary to messenger RNA (mRNA) to prevent specific proteins from being synthesized. When introduced into cells, these antisense oligonucleotides bind to their target mRNA through Watson-Crick base pairing, forming RNA-DNA hybrids that are degraded by RNase H enzyme. This prevents translation and expression of the target protein. There are three generations of antisense oligonucleotides that have been developed with improved stability and targeting capabilities, including phosphorothioate, 2'-O-methyl RNA, and locked nucleic acid chemistries. Antisense technology has potential applications in treating diseases like cancer, viral infections, and genetic disorders.
Gene therapy
This document discusses gene therapy approaches including ex-vivo and in-vivo gene therapy. Ex-vivo gene therapy involves removing cells from the body, introducing therapeutic genes in culture, and reintroducing the modified cells. In-vivo gene therapy directly delivers therapeutic genes into target cells using viral or non-viral vectors. Examples of each approach are provided, such as using ex-vivo gene therapy to treat adenosine deaminase deficiency and in-vivo gene therapy to treat cystic fibrosis. In conclusion, while gene therapy has potential, it faces complexity and high costs, limiting current accessibility.
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hi.friends this is my first slide presentation which contain the information about the PERSONALIZED MEDICINES.this is the future medicinal treatment so,I hope you people like my presentation.
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.
VIRAL VECTORS FOR GENE TRANSFER
Recombinant viral vectors are genetic engineering tools commonly used for gene transfer purpose with high transfection efficiency and site specific gene insertion.
Gene therapy of genetic disorders like hepatitis, neuroblastoma, thalassemia
Gene therapy is the modern techniques of treatment of various diseases and disorders.
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.
Inactivating, or “knocking out,” a mutated gene that is functioning improperly.
The first approved gene therapy experiment occurred on September1990 in US, when Ashanti DeSilva was treated for ADA-SCID.
Under the direction of William French Anderson, at the National Institutes of Health (NIH),
Cell Therapy - Definitions and Classifications
This document discusses definitions, classifications, and trends related to cell therapy. It defines cell therapy as the administration of cells to the body for therapeutic purposes and regenerative medicine as replacing or regenerating tissues to restore function. Classifications of cell therapies include immunocellular vs regenerative approaches and autologous vs allogeneic cell sources. The trends show growing interest in immunocellular therapies and use of mesenchymal stem cells and adipose tissue-derived cells. Regulatory classifications of cell therapies vary between countries and agencies and certain cell therapies can be difficult to clearly define, creating potential confusion.
Personalized medicine ppt
This document discusses personalized medicine, which aims to provide the right treatment for each individual patient based on their genetic profile. It defines personalized medicine as tailoring medical treatment to each patient's characteristics, needs and preferences. The development of genomic sequencing allows for more precise treatment by understanding how genetic variations impact drug metabolism and response. Pharmacogenomics studies how DNA and RNA variations affect drug effectiveness. Implementing personalized medicine through genetic testing can help reduce disease burden by improving prevention, treatment and healthcare costs while minimizing risks.
Genomics and proteomics in drug discovery and development
This document discusses the role of genomics and proteomics in drug discovery and development. It explains that genomics and proteomics technologies can help identify new drug targets by comparing gene and protein expression between healthy and diseased cells. Proteomics in particular analyzes changes in protein levels and can quantify individual proteins using techniques like 2D gel electrophoresis and mass spectrometry. The integration of genomics and proteomics provides a more comprehensive understanding of biological systems and is improving the drug discovery process.
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Gene therapy - current status and future perspective
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Gene therapy of genetic disorders like hepatitis, neuroblastoma, thalassemia
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Chalenges 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 types of gene therapy: somatic cell therapy and germline therapy. Key challenges for gene therapy include successfully delivering genes to the right tissues and cells, overcoming the immune response, avoiding disrupting important genes in target cells, and developing commercially viable therapies especially for rare genetic disorders.
Gene therapy
Gene therapy aims to treat genetic disorders by replacing or inactivating mutated genes. It involves inserting healthy genes into patient's cells using viral vectors or physical methods to replace defective genes that cause diseases. Some diseases potentially treatable with gene therapy include cancer, AIDS, Parkinson's, Alzheimer's, heart diseases, and arthritis. However, gene therapy faces challenges like short-lived effects, immune responses, issues with viral vectors, and difficulties treating disorders caused by multiple genes.
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
Gene therapy holds promise for treating diseases like cancer, HIV, and genetic disorders. It works by inserting a normal gene to replace an abnormal one causing disease. Viruses are often used as vectors to deliver the new gene into cells. There are two main types: germline alters genes in reproductive cells and is unethical, while somatic only alters genes in other body cells. Gene therapy has had some success but also deaths, and faces ethical issues regarding cost, safety, and enhancement versus treatment of disease. It remains experimental but could help many if developed responsibly.
Gene Therapy
In 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 IN DERMATOLOGY.pptx
Gene therapy involves introducing correct genes into patient cells to treat diseases caused by faulty genes. It was first conceptualized in 1972 and the first FDA-approved trial was in 1990. There are two main types - germline therapy modifies heritable germ cells while somatic therapy modifies non-heritable body cells. Common targets for skin gene therapy are keratinocytes, melanocytes and fibroblasts, which can be modified to treat genetic skin disorders, cancers and infections. While promising, gene therapy faces challenges like transient gene expression, immune reactions and safety issues with viral vectors.
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
Gene therapy involves introducing normal genes into patients to compensate for mutated genes that cause disease. The first gene therapy trial treated a girl with severe combined immunodeficiency. While it initially strengthened her immune system, the effects only lasted a few months. Gene therapy shows promise for diseases caused by single gene defects like cystic fibrosis, but faces challenges like short-lived effects, immune responses, and safety issues. Continued research aims to address these challenges through techniques like RNA interference and improved gene delivery methods.
Gene therapy dr. asm
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.
Gene therapy
Gene therapy involves inserting normal genes into patients' cells to treat genetic diseases. There are two main types - germline therapy, which affects future generations, and somatic therapy, which only affects the patient. Gene therapy works by using modified viruses or non-viral methods to deliver normal genes to replace mutated ones. Some successful cases include treating blindness caused by a genetic eye disease and reducing Parkinson's disease symptoms. While offering hope for currently untreatable diseases, gene therapy faces challenges in delivery methods, costs, and ethical issues.
Biology unit 5 genetics genetic engineering notes
Genetic engineering involves altering the genetic code of an organism. There are several common techniques: selective breeding exploits natural genetic variation; gene therapy replaces a faulty gene with a normal one using modified viruses; cell transformation inserts foreign DNA into a cell's genome to create transgenic organisms; and cloning produces genetically identical copies from a single cell. While gene therapy has had limited success so far, transgenic plants and animals have been created to improve traits like disease resistance and nutrient content.
Gene therapy presentation
Gene therapy is a technique for correcting defective genes that cause disease. It works by inserting a normal gene to replace a missing or defective one. The first human gene therapy treatment was in 1990 for ADA deficiency. There are two main types - germline alters genes in sperm/eggs and is inheritable, while somatic only affects the patient. Delivery methods include viruses, electroporation, gene guns, and chemicals. Applications show promise for diseases like Parkinson's, Alzheimer's, cystic fibrosis and cancer. Current trends include improving viral vectors and combining gene and cell therapies.
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
Genetherapy
Gene therapy involves introducing normal genes into patients to replace abnormal genes that cause disease. Viruses are often used as vectors to deliver therapeutic genes to target cells. While promising for treating genetic disorders, gene therapy faces challenges like short-lived effects, immune responses, and difficulties targeting multi-gene disorders. It also raises ethical issues around heritable genetic modifications and access to expensive new treatments. Continued research aims to address these scientific and ethical considerations to realize gene therapy's potential.
Gene therapy
Gene therapy involves introducing genes into cells to treat or cure diseases. It works by correcting defective genes that cause illnesses. The first approved human gene therapy trial took place in 1990 in the US and treated a patient with ADA-SCID. There are two main types of gene therapy - somatic cell gene therapy, which treats genes in body cells but is not inherited, and germline gene therapy, which treats genes in reproductive cells and can be passed to offspring. Gene therapy approaches include in vivo delivery of genes directly into tissues and ex vivo therapy involving culturing and modifying cells outside the body before reinsertion. Viral and non-viral vectors are used to transport therapeutic genes into target cells.
Gene therapy by bdollar
The slides give general description of gene therapy and some applications, factors to be considered when applying this method, viral vectors and non viral vectors applications and finally some successses of gene therapy
Gene therapy .pptx
Gene therapy is the study of methods thrugh which gene can be transferred from ons cell to another which can aid in treating thousands of disorders
Gene therapy 1 (2).pptx
Gene therapy is the study of methods through which we can teansfer gene from one cell to other and cure thousands of disorders with its help .
So hello everyone this is Prachi Mehta
Mpharm 1st yr student at GCU , guwahati . Hope this ppt gives brief idea on gene therapy .
Assignment on Advantages and Disadvantages of Gene Therapy.pdf
Gene therapy offers both advantages and disadvantages. The key advantages are that it can potentially cure diseases when other medications have failed, only requires a single treatment, and has long-lasting effects that can be passed down to future generations. However, gene therapy is also extremely expensive, still experimental with much unknown, and carries safety risks such as potentially infecting cells or causing mutations that could lead to cancer or other illnesses. There are also ethical concerns around who decides what is an appropriate use of gene therapy and how it could impact views on human differences.
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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Assignment on Advantages and Disadvantages of Gene Therapy.pdf
Assignment on Advantages and Disadvantages of Gene Therapy.pdf
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Challenges faced in gene therapy
- 1. Presented By: Syed Dawood Shah Challenges Faced In Gene Therapy Mirpur University Of Science And Technology Azaad Kashmir Pakistan
- 2. What is Gene Therapy? The introduction of normal genes into cells in place of missing or defective ones in order to correct genetic disorders
- 3. Types Of Gene Therapy • There are two types of gene therapy: 1. Somatic cell gene therapy 2. germline therapy
- 4. Challenges Faced In Gene Therapy • We have faced following Challenges during Gene Therapy: 1.Gene delivery and activation 2.Immune response 3.Disrupting important genes in target cells 4.Commercial viability
- 5. Gene delivery and activation • During gene therapy gene must reach to its destination & it must be activated. • Delivering a gene to the wrong tissue would be inefficient, and it could cause health problems for the patient.
- 6. Immune response • Our immune systems is very good at fighting to Foreign molecules. • Immune response is the very important challenge during gene therapy. • This situation is overcome by using immunosuppressive drugs to temporarily suppress the immune system.
- 7. Disrupting important genes in target cells • Ideal Gene therapy is that an introduced gene will continue working for the rest of the patient's life. • When we introduce foreign gene it alters the target cell genome and introduce new challenges.
- 8. Commercial viability • Some diseases that can be treated with gene therapy, such as cancer, are much more common. • Many genetic disorders that can potentially be treated with gene therapy are extremely rare, some affecting just one person out of a million. • Some gene therapy including taking in clinical trials necessary for government approval which are very expensive.