Non viral delivery systems for gene therapy (1)
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This document summarizes non-viral gene delivery systems. It discusses chemical non-viral systems, including cationic liposome/micelle based systems and cationic polymer based systems. Cationic liposome/micelle based systems are effective but heterogeneous and can have stability issues. They have potential for widespread delivery but need to overcome aggregation, cellular entry rate, endosomal escape, and immune response challenges. Cationic polymer systems include simple polymers like poly-L-lysine but require assisting agents. More advanced polymer systems show improved DNA interaction, serum resistance, and gene delivery efficiency. Overall, non-viral systems are promising for gene therapy but require overcoming barriers to stability, delivery efficiency, and safety.
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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.
Non viral delivery in gene therapy
This document summarizes non-viral methods for gene delivery in gene therapy. It discusses six main non-viral approaches: (1) naked nucleic acids, (2) liposomes, (3) particle bombardment, (4) receptor-mediated uptake, (5) polymer-complexed DNA, and (6) encapsulated cells. These methods are seen as safer alternatives to viral vectors but have been relatively neglected because viruses are more efficient at gene delivery.
Gene trasfer (Non Viral Gene Therapy)
Gene Therapy
2) What is Gene Therapy
An approach of treating diseases by either modifying the expressions of an individual’s genes or correction of abnormal genes.
3) This can be accomplished by:
Replacing a mutated gene that causes disease with a healthy copy of the gene.
Inactivating, or “knocking out,” a mutated gene that is functioning improperly.
Introducing a new gene into the body to help fight a disease
4) NON VIRAL VECTOR SYSTEM
5) 1. 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.
6) 2. DNA MOLECULAR CONJUGATES
Commonly used synthetic conjugate is poly- L- lysine bound to specific target cell receptor.
Therapeutic DNA is then made to combine with the conjugate to form a complex.
It avoids lysosomal breakdown of DNA
7) 3. LIPOPLEXES
Lipid DNA complexes
DNA construct surrounded by artificial lipid layer.
Most of it gets degraded by lysosomes
8) 4. HUMAN ARTIFICIAL CHROMOSOME
Can carry a large DNA i.e., with one or more therapeutic genes
9) References :-
Biotechnology by U. Satyanarayana and U. Chakrapani, First edition, books and allied (p) Ltd,2005. Page no.213-226.
NPTEL-Bio Technology – Genetic Engineering and applications “MODULE 5-LECTURE 1 GENE TRANSFER TECHNIQUES : BIOLOGICAL METHODS .” Page no : 3-22.
Gene therapy
The sequencing of the human genome has been compared to putting a man on the moon, and it will certainly change health care, but the most important work lies ahead, in determining how to put the information to medical use. In this context, applications such as gene therapy are being explored. What was once seen as a science fiction dream is now becoming a real possibility.
Gene therapy is a new form of drug delivery that leads the patient's own cells to produce a therapeutic agent. It could potentially eliminate the need for repeated administration of proteins or drugs. Applications of gene therapy not only include rare inherited diseases but extend to common acquired disorders, including tumours (predominantly malignant melanoma) and haematological disorders, cardiovascular disease, and the acquired immunodeficiency syndrome. Gene therapy therefore could be a key element of medical practice in the future. Gene therapy is the insertion of genes into an individual's cells and tissues to treat a disease, and hereditary diseases in which a defective mutant allele is replaced with a functional one. Although the technology is still in its infancy, it has been used with some success. Antisense therapy is not strictly a form of gene therapy, but is a genetically-mediated therapy and is often considered together with other methods.
Gene
This document summarizes gene expression systems, including both nonviral and viral gene transfer methods. It defines gene expression as the process by which information in DNA is used to produce a functional gene product like a protein. Nonviral methods include injecting naked DNA, as well as using physical methods like electroporation or chemical methods like lipoplexes to enhance DNA delivery into cells. Viral vectors are more efficient at transferring genes into cells and work by infecting cells with modified viruses that deliver therapeutic genes. Common viral vectors discussed are adenoviral, retroviral, and adeno-associated viral vectors.
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
Novel approach to treat, cure or ultimately prevent a disease by changing the expression of a person’s genes
POTENTIAL TARGET DISEASES FOR GENE THERAPY
Gene therapy has potential to treat many genetic diseases by introducing normal genes into patients' cells to compensate for mutated genes. Some potential target diseases include:
1) Type 1 diabetes, by inserting a gene for insulin production regulated by glucose levels, allowing rats to maintain normal blood sugar for over 8 months.
2) Cancer, using oncogene inactivation to reduce cancer-causing proteins or cell-targeted suicide genes combined with prodrugs to selectively kill cancer cells.
3) Parkinson's disease, by delivering a gene for the inhibitory neurotransmitter GABA into the brain to reduce tremors.
4) X-linked severe combined immunodeficiency (SCID), commonly known as "bubble
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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.
Non viral delivery in gene therapy
This document summarizes non-viral methods for gene delivery in gene therapy. It discusses six main non-viral approaches: (1) naked nucleic acids, (2) liposomes, (3) particle bombardment, (4) receptor-mediated uptake, (5) polymer-complexed DNA, and (6) encapsulated cells. These methods are seen as safer alternatives to viral vectors but have been relatively neglected because viruses are more efficient at gene delivery.
Gene trasfer (Non Viral Gene Therapy)
Gene Therapy
2) What is Gene Therapy
An approach of treating diseases by either modifying the expressions of an individual’s genes or correction of abnormal genes.
3) This can be accomplished by:
Replacing a mutated gene that causes disease with a healthy copy of the gene.
Inactivating, or “knocking out,” a mutated gene that is functioning improperly.
Introducing a new gene into the body to help fight a disease
4) NON VIRAL VECTOR SYSTEM
5) 1. 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.
6) 2. DNA MOLECULAR CONJUGATES
Commonly used synthetic conjugate is poly- L- lysine bound to specific target cell receptor.
Therapeutic DNA is then made to combine with the conjugate to form a complex.
It avoids lysosomal breakdown of DNA
7) 3. LIPOPLEXES
Lipid DNA complexes
DNA construct surrounded by artificial lipid layer.
Most of it gets degraded by lysosomes
8) 4. HUMAN ARTIFICIAL CHROMOSOME
Can carry a large DNA i.e., with one or more therapeutic genes
9) References :-
Biotechnology by U. Satyanarayana and U. Chakrapani, First edition, books and allied (p) Ltd,2005. Page no.213-226.
NPTEL-Bio Technology – Genetic Engineering and applications “MODULE 5-LECTURE 1 GENE TRANSFER TECHNIQUES : BIOLOGICAL METHODS .” Page no : 3-22.
Gene therapy
The sequencing of the human genome has been compared to putting a man on the moon, and it will certainly change health care, but the most important work lies ahead, in determining how to put the information to medical use. In this context, applications such as gene therapy are being explored. What was once seen as a science fiction dream is now becoming a real possibility.
Gene therapy is a new form of drug delivery that leads the patient's own cells to produce a therapeutic agent. It could potentially eliminate the need for repeated administration of proteins or drugs. Applications of gene therapy not only include rare inherited diseases but extend to common acquired disorders, including tumours (predominantly malignant melanoma) and haematological disorders, cardiovascular disease, and the acquired immunodeficiency syndrome. Gene therapy therefore could be a key element of medical practice in the future. Gene therapy is the insertion of genes into an individual's cells and tissues to treat a disease, and hereditary diseases in which a defective mutant allele is replaced with a functional one. Although the technology is still in its infancy, it has been used with some success. Antisense therapy is not strictly a form of gene therapy, but is a genetically-mediated therapy and is often considered together with other methods.
Gene
This document summarizes gene expression systems, including both nonviral and viral gene transfer methods. It defines gene expression as the process by which information in DNA is used to produce a functional gene product like a protein. Nonviral methods include injecting naked DNA, as well as using physical methods like electroporation or chemical methods like lipoplexes to enhance DNA delivery into cells. Viral vectors are more efficient at transferring genes into cells and work by infecting cells with modified viruses that deliver therapeutic genes. Common viral vectors discussed are adenoviral, retroviral, and adeno-associated viral vectors.
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
Novel approach to treat, cure or ultimately prevent a disease by changing the expression of a person’s genes
POTENTIAL TARGET DISEASES FOR GENE THERAPY
Gene therapy has potential to treat many genetic diseases by introducing normal genes into patients' cells to compensate for mutated genes. Some potential target diseases include:
1) Type 1 diabetes, by inserting a gene for insulin production regulated by glucose levels, allowing rats to maintain normal blood sugar for over 8 months.
2) Cancer, using oncogene inactivation to reduce cancer-causing proteins or cell-targeted suicide genes combined with prodrugs to selectively kill cancer cells.
3) Parkinson's disease, by delivering a gene for the inhibitory neurotransmitter GABA into the brain to reduce tremors.
4) X-linked severe combined immunodeficiency (SCID), commonly known as "bubble
GENE THERAPY: PRINCIPLES, PROBLEMS AND PROSPECTS
This document summarizes a seminar presentation on gene therapy. It discusses the basic principles of genes and gene therapy, including how genes encode proteins and how mutations can cause disease. It describes the two main types of gene therapy - somatic and germline. It also outlines some viral vectors used, methods of gene transfer like ex vivo and in vivo, diseases that may be treatable with gene therapy, prospects of the approach, and concludes that further development is needed but gene therapy has potential to benefit humanity.
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Gene therapy involves inserting genes into cells to prevent, alleviate or cure diseases caused by genetic defects. There are two main types of gene therapy - somatic cell gene therapy, which targets non-reproductive cells, and germline cell gene therapy, which targets reproductive cells and can be inherited. There are two main approaches - ex vivo gene therapy, where cells are modified outside the body, and in vivo gene therapy, where genes are modified inside the body. Viral and non-viral vectors are used to deliver genes to cells. Gene therapy has shown promise in treating diseases like SCID, cystic fibrosis, and cancer. While theoretically a permanent cure, gene therapy still faces challenges and high costs that limit widespread use.
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Gene therapy involves introducing genetic material into cells to treat disease. It is used for inherited disorders, cancers, and infectious diseases. Viruses are commonly used as vectors to deliver therapeutic genes, though safety issues exist. Some early successes included treating severe combined immunodeficiency using retroviruses to deliver the ADA gene ex vivo. However, the first death in gene therapy occurred treating ornithine transcarbamylase deficiency using an adenovirus vector, highlighting the risks. Improved delivery methods like liposomes may help overcome current limitations and advance this promising approach.
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Gene therapy aims to manage and correct human diseases like cancer, AIDS, and inherited disorders. While promising advances have been made in DNA technology, the efficacy of gene therapy protocols has yet to be definitively proven due to insufficient understanding of vector-host interactions. Gene therapy works by delivering exogenous DNA using a viral vector to enable protein expression in target cells. However, barriers like the immune system and lysosomes can prevent successful gene transfer. While many clinical applications exist, such as for inherited monogenic disorders and infectious diseases, safety issues remain a challenge as evidenced by the death of the first patient to die from a gene therapy treatment.
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Gene therapy has implications for dentistry including treatment of salivary glands, autoimmune disorders like Sjogren's syndrome, pain management, and oral cancer. Viral vectors are commonly used to introduce therapeutic genes but pose safety risks, while nonviral methods are safer but less efficient. Gene therapy shows promise for treating salivary gland disorders by introducing genes to produce proteins in saliva, treating autoimmune diseases by suppressing inflammatory genes, and managing pain by introducing genes for opiate peptides. It is also being explored for oral cancer through gene addition, antisense RNA, immunotherapy, and suicide gene therapy. Overall, gene therapy represents a new innovative approach for treating many oral diseases.
Gene therapy
The document discusses gene therapy, which involves inserting normal genes into patients to replace abnormal genes that cause diseases. The first approved gene therapy experiment occurred in 1990 when a 4-year-old girl with severe combined immunodeficiency was treated. There are two main types of gene therapy - somatic cell gene therapy, which treats cells in the body but is not inherited, and germ line gene therapy, which treats eggs and sperm and can be inherited but has safety and ethical concerns. Viruses are commonly used as vectors to deliver therapeutic genes directly to tissues or cells can be removed and treated ex vivo before being returned to the body. While gene therapy holds promise, it also faces challenges and risks that require further research.
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This research proposal aims to use gene therapy to prevent the rejection of islet cell transplants in type 1 diabetes patients. The researcher hypothesizes that by adding an immunosuppressive gene to the transplanted islet cells using an adeno-associated viral vector, the immune system will not attack the cells and endogenous insulin production can occur without other immunosuppressive drugs. The proposal involves testing this method on AKITA mice with diabetes by transplanting islet cells containing the CD47 gene or no added gene, and monitoring blood sugar levels to compare effectiveness of the transplanted cells at regulating blood sugar. The researcher expects the mice receiving cells with the CD47 gene will show greater insulin production and better blood sugar control.
Gene therapy
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.
Suicide gene therapy
Suicide gene therapy is based on the delivery of a gene encoding a cytotoxic protein into tumor cells.
For this, there are two possible strategies:
1. Indirect gene therapy using enzyme-activated pro-drug, which allows the conversion of a pro-drug into a lethal drug into cells.
2. Direct gene therapy using a toxin gene, whose expression can change the stability of the cell membrane and reduce the viability of tumor cells, or correct mutated pro-apoptotic genes, generally tumor suppressor genes that in normal condition induce cell suicide.
Gene Therapy
Gene therapy involves inserting functional genes into patients' cells to treat genetic diseases. It was first proposed in the 1960s and attempted in the 1970s. The first approved human gene therapy trial took place in 1990, treating a girl with severe combined immunodeficiency. Since then, gene therapy has been used experimentally to treat various diseases including cancer, cystic fibrosis, and HIV. There are two main approaches - ex vivo, where cells are removed and modified before being returned to the body, and in vivo, where genes are directly inserted into target tissues. Viruses are often used as vectors to deliver therapeutic genes, with retroviruses commonly used for ex vivo therapy and adenoviruses for in vivo.
Gene therapy, brief overview
Gene therapy involves introducing functional genes into patients' cells to treat diseases caused by defective genes. It works by replacing mutated genes with healthy copies, inactivating mutated genes, or introducing new genes to fight diseases. The process involves identifying the defective gene, cloning the normal gene, selecting target cells, and inserting the functional gene into the host DNA using viruses to deliver the new gene. Engineered cells are then injected into patients so the functional gene can correct the disorder.
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 introducing genes into cells to treat or prevent disease. It works by delivering a therapeutic gene into a patient's target cells using a vector. This can replace an abnormal gene with a healthy one, inactivate a mutated gene, or introduce a new gene to fight disease. There are two main types - somatic gene therapy only affects treated cells while germline gene therapy results in permanent changes that can be inherited. Approaches include ex vivo gene therapy, which modifies cells outside the body before transplant, and in vivo gene therapy through direct delivery. While gene therapy holds promise to cure genetic diseases, it faces challenges like short-lived effects and safety risks.
Gene therapy
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.
GENE THERAPY
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Gene delivery and gene therapy . Gene delivery , Gene therapy .
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List of contents under this ppt :
{A} GENE THERAPY
(1) Definition
(2) Introduction
(3) History
(4) Ex-Vivo gene therapy
(5) In-Vivo gene therapy
(6) Germline gene therapy
(7) Advantages of gene therapy
(8) Disadvantages of gene therapy
(9) Potential target diseases for gene therapy
a. inherited disorders :- ADA SCID, Chronic granulomatous, Hemophelia
b. Cancer
{B} GENE DELIVERY
(1) Definition
(2) Introduction
(3) Types of vectors
a. Viral :- Retrovirus, Adenovirus, Adeno associated virus, Herps simplex virus
b. Non viral :-
Physical methods - Gene gun, Microinjection, Electroporation, Sonoporation
Chemical methods - Oligonucleotides, Lipoplexes, Polyplexes, Dendrimers, Nanoparticles.
Gene therapy case study
This document summarizes a study on the feasibility, safety, and efficacy of gene therapy for hepatocellular carcinoma (HCC) using herpes simplex virus thymidine kinase (HSV-TK) suicide gene therapy. 10 patients with advanced HCC received intratumoral injections of an adenoviral vector containing HSV-TK followed by the prodrug ganciclovir. The therapy was found to be feasible and safe. Higher doses of the vector were associated with greater transgene expression. One patient experienced long term tumor control for over 18 months with no additional therapy. The results suggest HSV-TK gene therapy may produce antitumor effects for HCC.
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GENE THERAPY: PRINCIPLES, PROBLEMS AND PROSPECTS
This document summarizes a seminar presentation on gene therapy. It discusses the basic principles of genes and gene therapy, including how genes encode proteins and how mutations can cause disease. It describes the two main types of gene therapy - somatic and germline. It also outlines some viral vectors used, methods of gene transfer like ex vivo and in vivo, diseases that may be treatable with gene therapy, prospects of the approach, and concludes that further development is needed but gene therapy has potential to benefit humanity.
Gene therapy
Gene therapy involves inserting genes into cells to prevent, alleviate or cure diseases caused by genetic defects. There are two main types of gene therapy - somatic cell gene therapy, which targets non-reproductive cells, and germline cell gene therapy, which targets reproductive cells and can be inherited. There are two main approaches - ex vivo gene therapy, where cells are modified outside the body, and in vivo gene therapy, where genes are modified inside the body. Viral and non-viral vectors are used to deliver genes to cells. Gene therapy has shown promise in treating diseases like SCID, cystic fibrosis, and cancer. While theoretically a permanent cure, gene therapy still faces challenges and high costs that limit widespread use.
Gene therapy Otolaryngology
This presentation discusses the role of Gene therapy in the management of otolaryngological disorders
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Gene therapy involves introducing genetic material into cells to treat disease. It is used for inherited disorders, cancers, and infectious diseases. Viruses are commonly used as vectors to deliver therapeutic genes, though safety issues exist. Some early successes included treating severe combined immunodeficiency using retroviruses to deliver the ADA gene ex vivo. However, the first death in gene therapy occurred treating ornithine transcarbamylase deficiency using an adenovirus vector, highlighting the risks. Improved delivery methods like liposomes may help overcome current limitations and advance this promising approach.
Gene therapy lecture_spring_2007
Gene therapy aims to manage and correct human diseases like cancer, AIDS, and inherited disorders. While promising advances have been made in DNA technology, the efficacy of gene therapy protocols has yet to be definitively proven due to insufficient understanding of vector-host interactions. Gene therapy works by delivering exogenous DNA using a viral vector to enable protein expression in target cells. However, barriers like the immune system and lysosomes can prevent successful gene transfer. While many clinical applications exist, such as for inherited monogenic disorders and infectious diseases, safety issues remain a challenge as evidenced by the death of the first patient to die from a gene therapy treatment.
Gene therapy in dentistry
Gene therapy has implications for dentistry including treatment of salivary glands, autoimmune disorders like Sjogren's syndrome, pain management, and oral cancer. Viral vectors are commonly used to introduce therapeutic genes but pose safety risks, while nonviral methods are safer but less efficient. Gene therapy shows promise for treating salivary gland disorders by introducing genes to produce proteins in saliva, treating autoimmune diseases by suppressing inflammatory genes, and managing pain by introducing genes for opiate peptides. It is also being explored for oral cancer through gene addition, antisense RNA, immunotherapy, and suicide gene therapy. Overall, gene therapy represents a new innovative approach for treating many oral diseases.
Gene therapy
The document discusses gene therapy, which involves inserting normal genes into patients to replace abnormal genes that cause diseases. The first approved gene therapy experiment occurred in 1990 when a 4-year-old girl with severe combined immunodeficiency was treated. There are two main types of gene therapy - somatic cell gene therapy, which treats cells in the body but is not inherited, and germ line gene therapy, which treats eggs and sperm and can be inherited but has safety and ethical concerns. Viruses are commonly used as vectors to deliver therapeutic genes directly to tissues or cells can be removed and treated ex vivo before being returned to the body. While gene therapy holds promise, it also faces challenges and risks that require further research.
Diagnosis of inherited disorders and infectious disorders
Inherited disorders can be caused by genetic abnormalities passed down from parents or spontaneous genetic mutations. Diagnosis techniques include DNA probes tagged with radioactive isotopes to detect mutations, restriction enzyme analysis to study DNA fragments, and analysis of disease-related genes using PCR and fluorescent in situ hybridization. Examples provided are diagnosis of sickle cell anemia, cystic fibrosis, tuberculosis, cancer, and Down syndrome.
The Use of Gene Therapy to Treat Type 1 Diabetes Mellitus
This research proposal aims to use gene therapy to prevent the rejection of islet cell transplants in type 1 diabetes patients. The researcher hypothesizes that by adding an immunosuppressive gene to the transplanted islet cells using an adeno-associated viral vector, the immune system will not attack the cells and endogenous insulin production can occur without other immunosuppressive drugs. The proposal involves testing this method on AKITA mice with diabetes by transplanting islet cells containing the CD47 gene or no added gene, and monitoring blood sugar levels to compare effectiveness of the transplanted cells at regulating blood sugar. The researcher expects the mice receiving cells with the CD47 gene will show greater insulin production and better blood sugar control.
Gene therapy
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.
Suicide gene therapy
Suicide gene therapy is based on the delivery of a gene encoding a cytotoxic protein into tumor cells.
For this, there are two possible strategies:
1. Indirect gene therapy using enzyme-activated pro-drug, which allows the conversion of a pro-drug into a lethal drug into cells.
2. Direct gene therapy using a toxin gene, whose expression can change the stability of the cell membrane and reduce the viability of tumor cells, or correct mutated pro-apoptotic genes, generally tumor suppressor genes that in normal condition induce cell suicide.
Gene Therapy
Gene therapy involves inserting functional genes into patients' cells to treat genetic diseases. It was first proposed in the 1960s and attempted in the 1970s. The first approved human gene therapy trial took place in 1990, treating a girl with severe combined immunodeficiency. Since then, gene therapy has been used experimentally to treat various diseases including cancer, cystic fibrosis, and HIV. There are two main approaches - ex vivo, where cells are removed and modified before being returned to the body, and in vivo, where genes are directly inserted into target tissues. Viruses are often used as vectors to deliver therapeutic genes, with retroviruses commonly used for ex vivo therapy and adenoviruses for in vivo.
Gene therapy, brief overview
Gene therapy involves introducing functional genes into patients' cells to treat diseases caused by defective genes. It works by replacing mutated genes with healthy copies, inactivating mutated genes, or introducing new genes to fight diseases. The process involves identifying the defective gene, cloning the normal gene, selecting target cells, and inserting the functional gene into the host DNA using viruses to deliver the new gene. Engineered cells are then injected into patients so the functional gene can correct the disorder.
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 introducing genes into cells to treat or prevent disease. It works by delivering a therapeutic gene into a patient's target cells using a vector. This can replace an abnormal gene with a healthy one, inactivate a mutated gene, or introduce a new gene to fight disease. There are two main types - somatic gene therapy only affects treated cells while germline gene therapy results in permanent changes that can be inherited. Approaches include ex vivo gene therapy, which modifies cells outside the body before transplant, and in vivo gene therapy through direct delivery. While gene therapy holds promise to cure genetic diseases, it faces challenges like short-lived effects and safety risks.
Gene therapy
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.
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 rx [autosaved]
Gene therapy involves introducing genetic material into cells to treat or prevent disease. It has the potential to cure genetic disorders by correcting the underlying genetic defect. There are two main types - somatic gene therapy, which affects only targeted cells and is safer, and germline gene therapy which can permanently alter the genes and be passed to offspring. Recent advances include FDA-approved CAR-T immunotherapies for cancer and the first gene therapy approved for an inherited retinal disease. Challenges remain regarding delivery methods, safety, and ethical issues.
Gene delivery and gene therapy . Gene delivery , Gene therapy .
A good comprehensive review of gene delivery and gene therapy. especially for master of pharmacy 2nd-semester students as per the PCI syllabus of subject Molecular pharmaceutics.
List of contents under this ppt :
{A} GENE THERAPY
(1) Definition
(2) Introduction
(3) History
(4) Ex-Vivo gene therapy
(5) In-Vivo gene therapy
(6) Germline gene therapy
(7) Advantages of gene therapy
(8) Disadvantages of gene therapy
(9) Potential target diseases for gene therapy
a. inherited disorders :- ADA SCID, Chronic granulomatous, Hemophelia
b. Cancer
{B} GENE DELIVERY
(1) Definition
(2) Introduction
(3) Types of vectors
a. Viral :- Retrovirus, Adenovirus, Adeno associated virus, Herps simplex virus
b. Non viral :-
Physical methods - Gene gun, Microinjection, Electroporation, Sonoporation
Chemical methods - Oligonucleotides, Lipoplexes, Polyplexes, Dendrimers, Nanoparticles.
Gene therapy case study
This document summarizes a study on the feasibility, safety, and efficacy of gene therapy for hepatocellular carcinoma (HCC) using herpes simplex virus thymidine kinase (HSV-TK) suicide gene therapy. 10 patients with advanced HCC received intratumoral injections of an adenoviral vector containing HSV-TK followed by the prodrug ganciclovir. The therapy was found to be feasible and safe. Higher doses of the vector were associated with greater transgene expression. One patient experienced long term tumor control for over 18 months with no additional therapy. The results suggest HSV-TK gene therapy may produce antitumor effects for HCC.
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The Use of Gene Therapy to Treat Type 1 Diabetes Mellitus
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Gene delivery and gene therapy . Gene delivery , Gene therapy .
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Non viral delivery systems for gene therapy (1)
- 1. NON VIRAL DELIVERY SYSTEMS FOR GENE THERAPY
- 2. INTRODUCTION • Non viral vs viral systems • Encompass chemical and physical methods of DNA delivery • Promising future option for gene therapy
- 3. CHEMICAL NON VIRAL DELIVERY SYSTEMS: PRINCIPLES • Polycationic entities cause compaction of –vely charged nucleic acids forming nanometric complexes • Cationic liposome/micelle based: lipoplexes • Cationic polymer based: polyplexes • Protect bound NA • Have a net +ve charge • Enter cell by endocytosis by non specific interactions between complex and proteoglycans on adherent cells • Following entry, DNA can escape endosome and perform its function
- 4. • Instability towards aggregation under physiological salt conditions • Unstable in the presence of body fluids • Heterogeneous and polydisperse • Transfection takes long time • Inability to escape endosome • Difficulty to access nucleus CHEMICAL NON VIRAL DELIVERY SYSTEMS: PROBLEMS Efforts to develop many cationic entities No effort to develop one with all properties
- 6. CATIONIC LIPOSOME/MICELLE BASED NON VIRAL DELIVERY SYSTEMS • Most effective chemical NVDSs • Formed from • A single synthetic cationic amphiphile (cytofectin) • Combination of a cytofection and a neutral lipid (e.g., DOPE) • Currently >30 lipoplex systems • Cytofectin is the key component • Induction to unilamellar vesicles (cytofection+lipid) • Assembly into micellar structures (single cytofection) • Combining the above with DNA • Delivery to the cell
- 8. • Optimum in vivo delivery when the mol ratio of cationic liposome to DNA is such that the +ve to –ve charge ratio is 1. • Heterogeneous and polydisperse • Multilamellar complexes with DNA on the surface • Thin lipid coated DNA strands • Free nucleic acids • Which actually delivers DNA? CATIONIC LIPOSOME/MICELLE BASED NON VIRAL DELIVERY SYSTEMS
- 9. • In vitro transfection efficiency is a poor indicator of in vivo transfection efficiency • Less in vivo stability • Development of poly(ethylene)glycol-lipid conjugates • Use of cholesterol as lipid part • Widespread systemic delivery • Need for direct delivery but susceptibility to aggregation • Use of ligand modified lipoplex systems • Limited cell entry rate • Use of ligand modified lipoplex systems • Competition between specific natural ligand and non specific lipoplex CATIONIC LIPOSOME/MICELLE BASED NON VIRAL DELIVERY SYSTEMS
- 10. • Serum inactivation • Increasing +ve to –ve charge ratio • Introducing a lipoplex time dependent maturation • Immune and inflammatory responses • Use of stabilizing agents • Low rate of endosome escape • Use of low pH activated membrane active peptides • Use of pH sensitive liposomes • Development of hybrid liposome complexes CATIONIC LIPOSOME/MICELLE BASED NON VIRAL DELIVERY SYSTEMS
- 12. • Less widely used than cationic polymer based non viral delivery systems • Developed in the same time period • Two types • Simple polymers • Polymer systems CATIONIC POLYMER BASED NON VIRAL DELIVERY SYSTEMS
- 13. SIMPLE POLYMERS • Use of poly-L-lysine (pLL), a linear polymer • Can be produced in different mol.wt (19-1116 aa) • Conflict between optimal sizes for NA condensation and for gene delivery • Need of an assisting agent for efficient gene delivery • Use of EGF peptide or transferrin for facilitating cellular uptake • Less endosome escape • Use of fusogenic peptides or defective viral particles • Generally prone to aggregation • Too much additions needed for efficient output
- 14. • APL polycat-57: glucaramide nonpeptide, nonlipid polymer • Rapid interaction with plasmid DNA • Resistant to serum inhibition • Efficient gene delivery • Poly(N-ethyl-4-vinylpyridinium bromide, PEVP) • Copolymers • Peptoid polymers • Using DNA as template for polymer generation POLYMERS SYSTEMS