This document provides an overview of gene therapy and various gene transfer techniques. It discusses that gene therapy uses genes to treat or prevent disease by inserting genes into patient's cells. There are two main types of gene transfer techniques - non-viral and viral delivery systems. Non-viral techniques include physical methods like gene guns and electroporation, as well as chemical methods like cationic liposomes and polymers. Viral vectors are commonly used due to their efficiency and include retroviruses, adenoviruses, and adeno-associated viruses. The document reviews several applications of these various gene transfer techniques and concludes that while progress has been made, more development is still needed to design a safe and effective delivery system that can be
Liposomal gene delivery systems use liposomes to transport genetic material into cells. Liposomes are spherical lipid bilayers that can fuse with cell membranes. There are three types of lipids used - cationic lipids that bind to DNA, neutral lipids that stabilize the complex, and anionic lipids that are less efficient for delivery. Cationic liposomes form lipoplexes with DNA through electrostatic interactions and can transfect cells. Polyethylene glycol coating makes lipoplexes stealthier to avoid clearance. While liposomal systems provide nuclease protection and targeting, efficiency remains low and expression is transient. Research continues to improve targeting and stability for in vivo gene therapy applications.
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
This document provides an overview of gene expression and the central dogma of biology. It defines gene expression as the process by which genetic code is used to direct protein synthesis. The two main stages are transcription, where RNA is produced from DNA, and translation, where proteins are produced from mRNA. Key components of genes like exons, introns, promoters and enhancers are described. The details of transcription by RNA polymerase and the steps of translation by ribosomes are explained. The genetic code and how triplets of bases are used to represent the 20 amino acids is covered. The document concludes with an explanation of the central dogma that information flows from DNA to RNA to protein.
This document provides an overview of therapeutic aptamers. It defines aptamers as oligonucleotide molecules that bind to specific target molecules. Aptamers are produced using SELEX to select sequences with high affinity for target proteins. They have various therapeutic applications, such as inhibiting thrombin formation, amyloid-β propagation in Alzheimer's, and HIV integrase enzyme. Aptamers can also be used for targeted drug delivery by conjugating drugs to aptamers that bind cell surface proteins like nucleolin. The document discusses aptamer structure, production, modifications to improve stability, and advantages for therapeutic use.
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.
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.
The document discusses aptamers, which are single-stranded folded oligonucleotides or peptides that bind to molecular targets with high affinity and specificity. Aptamers are produced through an in vitro selection process called SELEX that identifies nucleic acid sequences that bind to a target. The document outlines the SELEX process and compares properties of aptamers to antibodies. Potential applications of aptamers discussed include use as therapeutics, drug delivery agents, diagnostic tools, and in bioimaging and Western blot analysis due to their high specificity and low immunogenicity.
This document provides an overview of gene therapy and various gene transfer techniques. It discusses that gene therapy uses genes to treat or prevent disease by inserting genes into patient's cells. There are two main types of gene transfer techniques - non-viral and viral delivery systems. Non-viral techniques include physical methods like gene guns and electroporation, as well as chemical methods like cationic liposomes and polymers. Viral vectors are commonly used due to their efficiency and include retroviruses, adenoviruses, and adeno-associated viruses. The document reviews several applications of these various gene transfer techniques and concludes that while progress has been made, more development is still needed to design a safe and effective delivery system that can be
Liposomal gene delivery systems use liposomes to transport genetic material into cells. Liposomes are spherical lipid bilayers that can fuse with cell membranes. There are three types of lipids used - cationic lipids that bind to DNA, neutral lipids that stabilize the complex, and anionic lipids that are less efficient for delivery. Cationic liposomes form lipoplexes with DNA through electrostatic interactions and can transfect cells. Polyethylene glycol coating makes lipoplexes stealthier to avoid clearance. While liposomal systems provide nuclease protection and targeting, efficiency remains low and expression is transient. Research continues to improve targeting and stability for in vivo gene therapy applications.
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
This document provides an overview of gene expression and the central dogma of biology. It defines gene expression as the process by which genetic code is used to direct protein synthesis. The two main stages are transcription, where RNA is produced from DNA, and translation, where proteins are produced from mRNA. Key components of genes like exons, introns, promoters and enhancers are described. The details of transcription by RNA polymerase and the steps of translation by ribosomes are explained. The genetic code and how triplets of bases are used to represent the 20 amino acids is covered. The document concludes with an explanation of the central dogma that information flows from DNA to RNA to protein.
This document provides an overview of therapeutic aptamers. It defines aptamers as oligonucleotide molecules that bind to specific target molecules. Aptamers are produced using SELEX to select sequences with high affinity for target proteins. They have various therapeutic applications, such as inhibiting thrombin formation, amyloid-β propagation in Alzheimer's, and HIV integrase enzyme. Aptamers can also be used for targeted drug delivery by conjugating drugs to aptamers that bind cell surface proteins like nucleolin. The document discusses aptamer structure, production, modifications to improve stability, and advantages for therapeutic use.
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.
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.
The document discusses aptamers, which are single-stranded folded oligonucleotides or peptides that bind to molecular targets with high affinity and specificity. Aptamers are produced through an in vitro selection process called SELEX that identifies nucleic acid sequences that bind to a target. The document outlines the SELEX process and compares properties of aptamers to antibodies. Potential applications of aptamers discussed include use as therapeutics, drug delivery agents, diagnostic tools, and in bioimaging and Western blot analysis due to their high specificity and low immunogenicity.
Liposomal gene delivery is an important non-viral gene delivery method. Cationic liposomes are used because the cationic lipid molecules can bind to the negatively charged DNA via electrostatic interactions. This forms lipoplexes that can enter cells via fusion or endocytosis. Liposomal gene delivery offers advantages over viral vectors like lower toxicity, but has lower gene transfer efficiency. It has potential applications in treating diseases by fixing or replacing defective genes.
Nucleic Acid Based Therapeutic Delivery System.pptxRAHUL PAL
Therapeutic nucleic acids (TNAs) are nucleic acids themselves or closely related compounds used to treat disease. Although various types of TNAs exist, they share a common mechanism of action that is mediated by sequence‐specific recognition of endogenous nucleic acids through Watson–Crick base pairing 7.
What are the advantages of nucleic acid based therapeutics?
The major advantage of nucleic acid-based therapeutics lies in the fact that they can be used to accurately target a tumor or tissue, then have a specific therapeutic protein, biologic, or immune engager expressed only at the site of interest.
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 involves inserting a normal gene to replace an abnormal gene that causes a genetic disease. It can replace or inactivate mutated genes, or introduce new genes to fight disease. Common applications include treating cystic fibrosis, hemophilia, cancer, and HIV. Viral vectors like retroviruses and adenoviruses are often used to deliver genes, but they can cause immune reactions. Non-viral methods like nanoparticles, electroporation, and ultrasound show promise for safer gene delivery. Overall, gene therapy holds potential for treating many currently incurable genetic disorders and diseases.
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 delivery is the process of introducing foreign DNA into host cells. There are four principal mechanisms for transferring genes into animal cells: direct physical transfer through microinjection or particle bombardment; chemical-mediated transfection using calcium phosphate or liposomes; transduction using viral vectors; and bactofection using bacterial vectors. Mammalian cells are widely used hosts as they allow production of recombinant human proteins with authentic post-translational modifications. Important applications include gene therapy and producing therapeutic proteins.
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.
Pharmacokinetics and pharmacodynamics of Biotechnological drugs-SnehalTidke
Pharmacokinetics and pharmacodynamics of biotechnological drugs along with appliations- Proteins and peptides, monoclonal antibodies, oligonucleotides, gene therapy and vaccines
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.
This document discusses biopharmaceuticals, which are medical drugs produced using biotechnology. It classifies biopharmaceuticals into several categories, including monoclonal antibodies, vaccines, thrombotic agents, interferons, blood factors, and hormones. Interferons are proteins produced by immune cells in response to challenges from viruses and tumors. They help the immune response by inhibiting viral replication and activating immune cells. Interferons are commercially produced from lymphocytes isolated from blood and induced to produce interferons. Biopharmaceuticals have advantages like being highly effective, specific, and safer than other drugs, with fewer side effects. They have many commercial applications in treating conditions like anemia, cancers, hepatitis B, and more. Biopharmaceuticals
Nucleic acid based therapeutic drug delivery systemtadisriteja9
Nucleic acid based Drug delivery system is one of the trending research area, which i have taken and made as Powerpoint for easy and quick learning purpose
This document provides an overview of population modelling as used in drug development. It discusses:
- The history and introduction of population modelling in 1972 to integrate data and aid drug development decisions.
- The types of models used, including PK, PKPD, disease progression, and meta-models.
- The components of population models, which include structural models describing response over time, stochastic models of variability, and covariate models of influencing factors.
- The concepts of model parameter estimation from data and model simulation to generate new data for evaluation and inference.
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.
Pharmacokinetics And Pharmacodynamic of Biotechnology Drugs - Trilok ShahareTrilok Shahare
This document discusses the pharmacokinetics and pharmacodynamics of biotechnology drugs. It begins by defining pharmacokinetics as the study of what the body does to a drug and pharmacodynamics as the study of a drug's biochemical and physiological effects. The document then examines various types of biotechnology products including proteins and peptides, monoclonal antibodies, oligonucleotides, vaccines, and gene therapies. It provides examples and applications of each type of biotechnology drug.
GENE THERAPY: TYPES, METHODS, FACTORS AND STANDARDS AND ITS APPLICATION IN HEALTHCARE FIELD
INVIVO THERAPY AND EXVIVO THERAPY
CHEMICAL AND PHYSICAL METHODS TO CARRY ON GENE THERAPY
DEFECTIVE GENE IDENTIFICATION IN GENE THERAPY AND TREATMENT OF GENETICALLY AFFECTED GENE BY GENE THERAPY
Gene therapy : Types, Gene transfer methods vectors for gene therapy approach...Shivkumar Sammeta
Gene therapy: Types of Gene therapy Gene transfer methods vectors for gene therapy approaches applications advantages and disadvantages. Gene therapy based drugs. Ethical considerations.
Liposomal gene delivery is an important non-viral gene delivery method. Cationic liposomes are used because the cationic lipid molecules can bind to the negatively charged DNA via electrostatic interactions. This forms lipoplexes that can enter cells via fusion or endocytosis. Liposomal gene delivery offers advantages over viral vectors like lower toxicity, but has lower gene transfer efficiency. It has potential applications in treating diseases by fixing or replacing defective genes.
Nucleic Acid Based Therapeutic Delivery System.pptxRAHUL PAL
Therapeutic nucleic acids (TNAs) are nucleic acids themselves or closely related compounds used to treat disease. Although various types of TNAs exist, they share a common mechanism of action that is mediated by sequence‐specific recognition of endogenous nucleic acids through Watson–Crick base pairing 7.
What are the advantages of nucleic acid based therapeutics?
The major advantage of nucleic acid-based therapeutics lies in the fact that they can be used to accurately target a tumor or tissue, then have a specific therapeutic protein, biologic, or immune engager expressed only at the site of interest.
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 involves inserting a normal gene to replace an abnormal gene that causes a genetic disease. It can replace or inactivate mutated genes, or introduce new genes to fight disease. Common applications include treating cystic fibrosis, hemophilia, cancer, and HIV. Viral vectors like retroviruses and adenoviruses are often used to deliver genes, but they can cause immune reactions. Non-viral methods like nanoparticles, electroporation, and ultrasound show promise for safer gene delivery. Overall, gene therapy holds potential for treating many currently incurable genetic disorders and diseases.
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 delivery is the process of introducing foreign DNA into host cells. There are four principal mechanisms for transferring genes into animal cells: direct physical transfer through microinjection or particle bombardment; chemical-mediated transfection using calcium phosphate or liposomes; transduction using viral vectors; and bactofection using bacterial vectors. Mammalian cells are widely used hosts as they allow production of recombinant human proteins with authentic post-translational modifications. Important applications include gene therapy and producing therapeutic proteins.
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.
Pharmacokinetics and pharmacodynamics of Biotechnological drugs-SnehalTidke
Pharmacokinetics and pharmacodynamics of biotechnological drugs along with appliations- Proteins and peptides, monoclonal antibodies, oligonucleotides, gene therapy and vaccines
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.
This document discusses biopharmaceuticals, which are medical drugs produced using biotechnology. It classifies biopharmaceuticals into several categories, including monoclonal antibodies, vaccines, thrombotic agents, interferons, blood factors, and hormones. Interferons are proteins produced by immune cells in response to challenges from viruses and tumors. They help the immune response by inhibiting viral replication and activating immune cells. Interferons are commercially produced from lymphocytes isolated from blood and induced to produce interferons. Biopharmaceuticals have advantages like being highly effective, specific, and safer than other drugs, with fewer side effects. They have many commercial applications in treating conditions like anemia, cancers, hepatitis B, and more. Biopharmaceuticals
Nucleic acid based therapeutic drug delivery systemtadisriteja9
Nucleic acid based Drug delivery system is one of the trending research area, which i have taken and made as Powerpoint for easy and quick learning purpose
This document provides an overview of population modelling as used in drug development. It discusses:
- The history and introduction of population modelling in 1972 to integrate data and aid drug development decisions.
- The types of models used, including PK, PKPD, disease progression, and meta-models.
- The components of population models, which include structural models describing response over time, stochastic models of variability, and covariate models of influencing factors.
- The concepts of model parameter estimation from data and model simulation to generate new data for evaluation and inference.
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.
Pharmacokinetics And Pharmacodynamic of Biotechnology Drugs - Trilok ShahareTrilok Shahare
This document discusses the pharmacokinetics and pharmacodynamics of biotechnology drugs. It begins by defining pharmacokinetics as the study of what the body does to a drug and pharmacodynamics as the study of a drug's biochemical and physiological effects. The document then examines various types of biotechnology products including proteins and peptides, monoclonal antibodies, oligonucleotides, vaccines, and gene therapies. It provides examples and applications of each type of biotechnology drug.
GENE THERAPY: TYPES, METHODS, FACTORS AND STANDARDS AND ITS APPLICATION IN HEALTHCARE FIELD
INVIVO THERAPY AND EXVIVO THERAPY
CHEMICAL AND PHYSICAL METHODS TO CARRY ON GENE THERAPY
DEFECTIVE GENE IDENTIFICATION IN GENE THERAPY AND TREATMENT OF GENETICALLY AFFECTED GENE BY GENE THERAPY
Gene therapy : Types, Gene transfer methods vectors for gene therapy approach...Shivkumar Sammeta
Gene therapy: Types of Gene therapy Gene transfer methods vectors for gene therapy approaches applications advantages and disadvantages. Gene therapy based drugs. Ethical considerations.
This document discusses viral vectors, which are tools used by molecular biologists to deliver genetic material into cells. It describes how viruses are efficient at transferring their DNA into host cells and can be modified to insert exogenous genes. The document outlines some key properties of viral vectors like safety through deletion of viral replication genes. It then describes some common viral vectors, including adenovirus, adeno-associated virus, herpes virus, lentivirus, and retrovirus. Finally, it discusses some applications of viral vectors like gene therapy and using viruses expressing pathogen proteins as vaccines.
Nucleic acid and cell based therapies involve gene therapy and cell therapy. Gene therapy aims to introduce new genes into cells to treat genetic diseases by replacing defective genes. Early gene therapy trials focused on ex vivo and in vivo approaches. Viral vectors like retroviruses were primarily used but posed safety risks. Non-viral methods using naked DNA or vectors like liposomes were developed. Diseases targeted include cancer, genetic disorders, and AIDS. Antisense oligonucleotides can bind mRNA and inhibit gene expression. RNA interference uses short interfering RNAs to induce sequence-specific gene silencing. Ribozymes and aptamers also provide nucleic acid based therapeutic approaches.
Gene therapy involves techniques that modify or manipulate genes to treat or prevent diseases. The first gene therapy treatment occurred in 1990 for severe combined immunodeficiency. There are four main approaches to gene therapy: inserting a normal gene to compensate for a defective one, replacing an abnormal gene with a normal one, repairing an abnormal gene, or altering gene regulation. Viruses are commonly used as vectors to deliver therapeutic genes into target cells, with retroviruses, adenoviruses, adeno-associated viruses, and herpes simplex viruses being some of the most widely used viral vectors, each with advantages and limitations.
Gene therapy with viral and non viral vectors.pptxaditi276464
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.
This document discusses gene transfer techniques, including viral and non-viral delivery systems. It describes gene therapy as using genes to treat disease by inserting a gene into a patient's cells instead of using drugs or surgery. It outlines various non-viral physical methods like gene guns, ultrasound, electroporation, and magnetofection and chemical methods like cationic liposomes and polymers to facilitate gene transfer. Viral vectors discussed include retroviruses, adenoviruses, and adeno-associated viruses. In conclusion, while progress has been made, developing safe and effective non-viral delivery systems for in vivo gene therapy remains a challenge.
On January 25, 2022, Nature published an article listing seven technologies worthy of attention this year. Targeted genetic therapies was on the list. The remaining six technologies are: Fully finished genomes, Protein structure solutions, Quantum simulation, Precise genome manipulation, Spatial multi-omics), CRISPR-based diagnostics.
This document discusses gene therapy and its potential to treat diseases. It provides an overview of genes and how mutations can cause disease. Gene therapy involves inserting functional genes into patients' cells to replace mutated genes that cause illness. Viruses called vectors are used to deliver therapeutic genes into target cells. Some common vector types discussed are retroviruses, adenoviruses, and adeno-associated viruses. Both ex vivo and in vivo gene therapy approaches are described. The document reviews the history of gene therapy and various clinical trials that have been conducted to treat diseases like cancer, cystic fibrosis, and immunodeficiencies.
This document discusses adenoviral cloning vectors. It begins by defining a cloning vector as a small piece of DNA that can be stably maintained in an organism and have foreign DNA inserted into it for cloning purposes. It then discusses viral vectors, noting that they are commonly used to deliver genetic material into cells through transduction. The document focuses on properties of viral vectors, specifically safety features and targeting abilities. It provides details on adenoviruses, noting they can efficiently transfer genes, their structure, applications in gene therapy and vaccination, and their DNA genome capacity. Adeno-associated viruses are also mentioned as attractive for gene therapy due to mild immune response.
Gene therapy involves delivering genetic material to cells to alter their instructions for a therapeutic purpose. The first human gene therapy trial was conducted in 1990, though no gene therapy products are commercially available yet. Key goals for gene therapy strategies are to administer treatments easily, achieve long-term therapeutic effects from a single application, and target effects specifically to diseased cells with few side effects. Choosing the right viral vector depends on factors like the target cells and desired gene expression level and duration.
The document discusses various viral delivery systems for gene therapy. It notes four major problems with current gene therapy: short-lived expression, immune response, issues with viral vectors, and difficulties treating multigene disorders. Common viral vectors discussed include retroviruses, adenoviruses, adeno-associated viruses, and herpes simplex viruses. Each vector type has advantages and limitations for gene delivery applications. Retroviruses can only target dividing cells, while lentiviruses can target both dividing and non-dividing cells. Adenoviral vectors have high transduction efficiency but also elicit strong immune responses. AAV vectors allow long-term expression but have a small cargo capacity.
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 refers to the insertion of genetic material to correct a genetic defect.
In gene therapy, a "normal" gene is inserted into the genome to replace an "abnormal," disease-causing gene
Gene therapy involves introducing genes into cells to treat or prevent disease. It works by correcting defective genes that cause disease or by making cells produce products to treat the disease. The first approved gene therapy treated a girl for ADA-SCID. There are two main approaches - in vivo therapy directly delivers genes into body cells, while ex vivo therapy transfers genes to cultured cells before reinsertion. Viral vectors like retroviruses and adenoviruses are often used due to their ability to deliver genes, but come with risks like insertional mutagenesis. Non-viral methods include physical methods like microinjection and chemical methods using liposomes. Gene therapy shows promise for diseases like cancer, cardiovascular disease, and neurological disorders.
Gene therapy involves introducing normal genes into patients to compensate for mutated genes that cause disease. It works by using a vector to deliver the therapeutic gene into a target cell, allowing functional proteins to be produced and returning the cell to a normal state. There are two main types - germline gene therapy, which can pass therapeutic effects to future generations, and somatic gene therapy, which only affects the individual patient. While initial gene therapy trials showed promise, there have also been safety issues, as in 1999 a patient died due to an immune response to the adenovirus vector. Researchers continue working to address risks before conducting further human clinical trials.
This document provides an overview of gene expression systems and vectors used for gene transfer. It discusses the key phases of gene expression including transcription, post-transcriptional modifications, RNA transport, translation, and protein binding. It describes the two major categories of gene therapy - somatic and germline - and the three types of delivery - ex vivo, in situ, and in vitro. Finally, it summarizes the major viral vectors including retroviruses, adenoviruses, lentiviruses, and adeno-associated viruses, as well as various non-viral physical methods like electroporation and chemical methods using inorganic particles and biodegradable polymers.
Penetration Enhancers in Transdermal Drug Delivery SystemSimranDhiman12
Penetration Enhancers in Transdermal Drug Delivery System
Permeation enhancers are substances that reduce the skin barrier's ability to make skin more permeable and allow drug molecules to cross the skin at a faster rate
advantages and disadvantages
types of penetration enhancers
techniques
physical and chemical enhancers
Regulatory requirements for API and BiologicsSimranDhiman12
The document discusses regulatory requirements for active pharmaceutical ingredients (APIs) and biologics. It provides an overview of regulatory guidelines for APIs, including requirements for registration of APIs with agencies like the FDA. It also describes regulatory filings like Drug Master Files (DMFs) that are submitted to provide confidential manufacturing information to support applications. Requirements for biologics are also briefly covered, noting they are complex molecules produced through biotechnology.
This document summarizes non-viral gene transfer techniques. It discusses that gene expression is the process by which genetic information is used to produce functional products like proteins. Gene therapy techniques use genes to treat diseases by delivering transgenes into cells. Non-viral methods of gene transfer include chemical methods like oligonucleotides, liposomes, lipoplexes, and polymersomes, as well as physical methods like gene guns, electroporation, sonoporation, magnetofection, and hydrodynamic delivery. These non-viral methods facilitate intracellular delivery of DNA through mechanisms like forming transient pores in cell membranes.
Perfumes, Classification, Perfume Ingredients listed as allergens in EU regul...SimranDhiman12
This document summarizes the key aspects of perfumes, including their composition, manufacturing process, classification, and ingredients. Perfumes are mixtures of essential oils, fixatives, and solvents that are blended according to a formula. They are classified based on concentration and longevity. The major steps in manufacturing include collection of aromatic sources, extraction of oils, blending, and aging. Perfumes contain top, middle, and base notes that create the harmonious scent. They also list common allergens that must be disclosed on cosmetic labels in the EU.
Inventory Management and Control, Production Planning and ControlSimranDhiman12
This document provides an overview of inventory management, production planning, and control. It discusses key objectives like minimizing costs and ensuring adequate supply. Inventory management techniques include ABC analysis, VED analysis, EOQ, lead time, and buffer stock. Production planning determines facility requirements and layout, while production control monitors plan execution and addresses deviations. The perpetual inventory system uses bin cards, store ledgers, and continuous stock taking to regularly check and prevent stockouts.
The document discusses the New Drug Application (NDA) process required by the FDA to approve new pharmaceutical drugs for sale and marketing in the United States. An NDA provides data from animal and human clinical trials to allow the FDA to determine if the drug is safe, effective and manufactured properly. It includes details on manufacturing, packaging, non-clinical and clinical testing results. If clinical trials confirm the drug's safety and effectiveness, manufacturers can file an NDA to request approval to produce and sell the drug. The NDA review process evaluates whether the drug's risks are outweighed by its benefits and whether labeling and quality controls are adequate.
This document defines and describes the role and responsibilities of an Institutional Review Board (IRB). The key points are:
1) An IRB is responsible for reviewing and approving research involving human subjects to ensure ethical standards are met and subjects' rights and safety are protected.
2) IRBs must have diverse and qualified membership, including scientific and non-scientific experts, to conduct in-depth reviews of research protocols.
3) The IRB review process involves evaluating research protocols, consent forms, recruitment procedures and investigators to ensure all applicable regulations and ethical guidelines are followed.
UV-Visible Spectroscopy is a technique that uses light in the visible and adjacent ranges. It works based on how molecules absorb light at specific wavelengths. The document discusses the principles of UV-Vis spectroscopy including Beer's law, electronic transitions, chromophores and auxochromes. It also covers instrumentation components like light sources, monochromators, sample cells and detectors. Factors affecting absorption spectra are solvents, pH and conjugation. The technique has applications in analytical chemistry for identification and quantification of analytes.
Does Over-Masturbation Contribute to Chronic Prostatitis.pptxwalterHu5
In some case, your chronic prostatitis may be related to over-masturbation. Generally, natural medicine Diuretic and Anti-inflammatory Pill can help mee get a cure.
Basavarajeeyam is a Sreshta Sangraha grantha (Compiled book ), written by Neelkanta kotturu Basavaraja Virachita. It contains 25 Prakaranas, First 24 Chapters related to Rogas& 25th to Rasadravyas.
Here is the updated list of Top Best Ayurvedic medicine for Gas and Indigestion and those are Gas-O-Go Syp for Dyspepsia | Lavizyme Syrup for Acidity | Yumzyme Hepatoprotective Capsules etc
ABDOMINAL TRAUMA in pediatrics part one.drhasanrajab
Abdominal trauma in pediatrics refers to injuries or damage to the abdominal organs in children. It can occur due to various causes such as falls, motor vehicle accidents, sports-related injuries, and physical abuse. Children are more vulnerable to abdominal trauma due to their unique anatomical and physiological characteristics. Signs and symptoms include abdominal pain, tenderness, distension, vomiting, and signs of shock. Diagnosis involves physical examination, imaging studies, and laboratory tests. Management depends on the severity and may involve conservative treatment or surgical intervention. Prevention is crucial in reducing the incidence of abdominal trauma in children.
Rasamanikya is a excellent preparation in the field of Rasashastra, it is used in various Kushtha Roga, Shwasa, Vicharchika, Bhagandara, Vatarakta, and Phiranga Roga. In this article Preparation& Comparative analytical profile for both Formulationon i.e Rasamanikya prepared by Kushmanda swarasa & Churnodhaka Shodita Haratala. The study aims to provide insights into the comparative efficacy and analytical aspects of these formulations for enhanced therapeutic outcomes.
Adhd Medication Shortage Uk - trinexpharmacy.comreignlana06
The UK is currently facing a Adhd Medication Shortage Uk, which has left many patients and their families grappling with uncertainty and frustration. ADHD, or Attention Deficit Hyperactivity Disorder, is a chronic condition that requires consistent medication to manage effectively. This shortage has highlighted the critical role these medications play in the daily lives of those affected by ADHD. Contact : +1 (747) 209 – 3649 E-mail : sales@trinexpharmacy.com
NVBDCP.pptx Nation vector borne disease control programSapna Thakur
NVBDCP was launched in 2003-2004 . Vector-Borne Disease: Disease that results from an infection transmitted to humans and other animals by blood-feeding arthropods, such as mosquitoes, ticks, and fleas. Examples of vector-borne diseases include Dengue fever, West Nile Virus, Lyme disease, and malaria.
These lecture slides, by Dr Sidra Arshad, offer a quick overview of the physiological basis of a normal electrocardiogram.
Learning objectives:
1. Define an electrocardiogram (ECG) and electrocardiography
2. Describe how dipoles generated by the heart produce the waveforms of the ECG
3. Describe the components of a normal electrocardiogram of a typical bipolar lead (limb II)
4. Differentiate between intervals and segments
5. Enlist some common indications for obtaining an ECG
6. Describe the flow of current around the heart during the cardiac cycle
7. Discuss the placement and polarity of the leads of electrocardiograph
8. Describe the normal electrocardiograms recorded from the limb leads and explain the physiological basis of the different records that are obtained
9. Define mean electrical vector (axis) of the heart and give the normal range
10. Define the mean QRS vector
11. Describe the axes of leads (hexagonal reference system)
12. Comprehend the vectorial analysis of the normal ECG
13. Determine the mean electrical axis of the ventricular QRS and appreciate the mean axis deviation
14. Explain the concepts of current of injury, J point, and their significance
Study Resources:
1. Chapter 11, Guyton and Hall Textbook of Medical Physiology, 14th edition
2. Chapter 9, Human Physiology - From Cells to Systems, Lauralee Sherwood, 9th edition
3. Chapter 29, Ganong’s Review of Medical Physiology, 26th edition
4. Electrocardiogram, StatPearls - https://www.ncbi.nlm.nih.gov/books/NBK549803/
5. ECG in Medical Practice by ABM Abdullah, 4th edition
6. Chapter 3, Cardiology Explained, https://www.ncbi.nlm.nih.gov/books/NBK2214/
7. ECG Basics, http://www.nataliescasebook.com/tag/e-c-g-basics
Muktapishti is a traditional Ayurvedic preparation made from Shoditha Mukta (Purified Pearl), is believed to help regulate thyroid function and reduce symptoms of hyperthyroidism due to its cooling and balancing properties. Clinical evidence on its efficacy remains limited, necessitating further research to validate its therapeutic benefits.
Recomendações da OMS sobre cuidados maternos e neonatais para uma experiência pós-natal positiva.
Em consonância com os ODS – Objetivos do Desenvolvimento Sustentável e a Estratégia Global para a Saúde das Mulheres, Crianças e Adolescentes, e aplicando uma abordagem baseada nos direitos humanos, os esforços de cuidados pós-natais devem expandir-se para além da cobertura e da simples sobrevivência, de modo a incluir cuidados de qualidade.
Estas diretrizes visam melhorar a qualidade dos cuidados pós-natais essenciais e de rotina prestados às mulheres e aos recém-nascidos, com o objetivo final de melhorar a saúde e o bem-estar materno e neonatal.
Uma “experiência pós-natal positiva” é um resultado importante para todas as mulheres que dão à luz e para os seus recém-nascidos, estabelecendo as bases para a melhoria da saúde e do bem-estar a curto e longo prazo. Uma experiência pós-natal positiva é definida como aquela em que as mulheres, pessoas que gestam, os recém-nascidos, os casais, os pais, os cuidadores e as famílias recebem informação consistente, garantia e apoio de profissionais de saúde motivados; e onde um sistema de saúde flexível e com recursos reconheça as necessidades das mulheres e dos bebês e respeite o seu contexto cultural.
Estas diretrizes consolidadas apresentam algumas recomendações novas e já bem fundamentadas sobre cuidados pós-natais de rotina para mulheres e neonatos que recebem cuidados no pós-parto em unidades de saúde ou na comunidade, independentemente dos recursos disponíveis.
É fornecido um conjunto abrangente de recomendações para cuidados durante o período puerperal, com ênfase nos cuidados essenciais que todas as mulheres e recém-nascidos devem receber, e com a devida atenção à qualidade dos cuidados; isto é, a entrega e a experiência do cuidado recebido. Estas diretrizes atualizam e ampliam as recomendações da OMS de 2014 sobre cuidados pós-natais da mãe e do recém-nascido e complementam as atuais diretrizes da OMS sobre a gestão de complicações pós-natais.
O estabelecimento da amamentação e o manejo das principais intercorrências é contemplada.
Recomendamos muito.
Vamos discutir essas recomendações no nosso curso de pós-graduação em Aleitamento no Instituto Ciclos.
Esta publicação só está disponível em inglês até o momento.
Prof. Marcus Renato de Carvalho
www.agostodourado.com
2. INTRODUCTION
GENE EXPRESSION
It is the process by which a gene’s DNA sequence is converted into the
structures and functions of a cell.
Non-protein coding genes are not translated into protein.
Gene expression refers to a complex series of processes in which the
information encoded in a gene is used to produce a functional product such
as a protein that dictates cell function.
Genetic information, chemically determined by DNA structure is transferred
to daughter cells by DNA replication and expressed by Transcription
followed by Translation.
3. This series of events is called “Central Dogma” is found in all cells and
proceeds in similar ways except in retroviruses which posses an enzyme
reverse transcriptase which converts RNA into complementary DNA.
Biological information flows from DNA to RNA, and from there to proteins.
4.
5. WHAT IS GENE THERAPY?
Gene therapy is an Experimental technique that uses genes to treat or prevent
disease.
Basically gene therapy is an intracellular delivery of genomic materials
(transgene) into specific cells to generate a therapeutic effect by correcting
an existing abnormality or providing the cells with a new function.
6. GENE TRANSFER
Gene transfer is the introduction of foreign genes or gene sequences in a target cell
population using nonviral or viral vectors to treat a particular disorder in affected
individual for the sustained expression of a transgene cassette vs application of the
therapeutic product itself that exhibits a short pharmacological half-life.
There are different reasons to do gene transfer. Perhaps foremost these reasons is the
treatment of diseases using gene transfer to supply patients with therapeutic genes.
There are different ways to transfer genes. Some of methods involve the use of a
vector such as a virus so it can take the gene along with it when it enters the cell.
7. GENE TRANSFER TECHNIQUES
Based on the vectors used the gene transfer techniques can be divided as
1. Viral methods
2. Non Viral methods
9. VECTOR AND IT’S IDEAL PROPERTIES
A Vector can be described as a system fulfilling several functions:
1. Enabling delivery of genes to target cells and their nucleus.
2. Providing protection from gene degradation.
3. Ensuring gene transcription in the cells.
10. VECTOR SYSTEM FOR GENE
DELIVERY
Viral vectors
Viruses are naturally evolved vehicles that efficiently transfer their genes into
host cells.
Choice of viral vector is dependent on gene transfer efficiency, capacity to carry
foreign genes, toxicity, stability. Immune responses towards viral antigens and
potential viral recombination.
One of the successful gene therapy systems available today are viral vectors,
such as retrovirus, adenovirus (types 2 and 5), adeno-associated virus,
herpes virus, pox virus, human foamy virus (HFV), and lentivirus.
All viral vector genomes have been modified by deleting some areas of their
genomes so that their replication becomes deranged and it makes them more
safe.
11.
12. RETROVIRAL VECTORS
Retroviral vectors are one of the most frequently employed forms of gene delivery in
somatic and germline gene therapies.
Commonly employed vectors derived from Murine Leukemia Virus (MuLV).
Virus genome has two single copy RNA molecules, complexed with viral core proteins,
surrounded by lipid envelope.
In addition, all of the viral genes have been removed.
For example, they have been used for human gene therapy of X-SCID successfully.
Applications: Ex Vivo gene therapy
In vivo gene transfer using retro viral vectors for suicide gene used in brain tumour
Treatment of T lymphocyte deficiency ,Tumour Infiltrating Lymphocyte(TIL), bone
marrow cells, Gauchers disease, hepatocytes(LDL receptor deficiency) and melanoma.
13.
14. ADENO VIRUS VECTORS
These are non enveloped DNA viruses, linear genome and double stranded DNA
molecule of about 36kb.
Adeno viral vectors have been isolated from a large number of different species and
more than 100 different serotypes have been reported
Adeno viruses type 2 and type 5 can be utilized the transferring both dividing and no
dividing cells and have low host specificity.
Application:
to vivo gene therapy- transduce non dividing and terminally differentiated cells.
Transfect cells in vivo in the intact organ
Gene therapy for cystic therapy
Gene therapy of muscle in liver and therapy of disease of CNS
Localized cancer gene therapy.
15. ADENO ASSOCIATED VIRUS VECTORS
Members of Parvovirus family
Heat stable and resistant to various chemicals
Depend on virus- cannot replicate its own, another virus is necessary for
replication
Major disadvantages of these vectors are complicated process of vector
production and the limited transgene capacity of the particles.
Applications
Used in hematopoietic stem cells for treatment of B-thalassemia and sickle
cell anemia
AAVs have been used in the treatment of some diseases, such as CF,
hemophilia B
16. HERPES SIMPLEX VIRUS
Herpes simplex virus (HSV) is one of the recent viruses candidate in gene
delivery.
When the defective HSV propagated in complementing cells’ viral particles
are generated, they can infect in subsequent cells permanently replicating
their own genome but not producing more infectious particles.
Herpes vectors can deliver up to 150 kb transgenic DNA and because of its
neuronotropic features, it has the greatest potential for gene delivery to
nervous system tumors, and cancer cells.
17. LENTIVIRUSES
Lentiviruses are a subclass of retroviruses. They have recently been used as
gene delivery vectors due to their ability to naturally integrate with
nondividing cells.
Lentiviral vectors can deliver 8 kb of sequence.
Used for ex vivo gene transfer in central nervous system with no significant
immune responses and no unwanted side effects.
Effective for long-term treatment of animal models of neurologic disorders,
such as motor neuron diseases, Parkinson, Alzheimer, Huntington’s disease,
lysosomal storage diseases, and spinal injury.