This document summarizes viral and non-viral gene transfer techniques. It discusses that gene therapy involves introducing genes into host cells to treat diseases. The two main methods are viral and non-viral. Non-viral techniques include physical methods like gene guns and electroporation, as well as chemical methods like cationic liposomes and polymers. Viral vectors commonly used are retroviruses, adenoviruses, and adeno-associated viruses. While viral vectors have higher efficiency, non-viral methods have advantages like lower cost and immune response. Overall, both methods show promise for gene therapy but further improving delivery systems is still needed.
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.
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
Introduction
Approaches to Gene Therapy
Vectors in Gene Therapy
Non-viral Methods
Physical Methods for Improving DNA Transfer
Chemical Methods for Improving DNA Transfer
Advantages and Disadvantages of Gene Therapy
Applications of Gene Therapy
Challenges
The document discusses gene therapy as a promising approach for treating various diseases. It provides a brief history of gene therapy and describes some of the early clinical trials. It then explains some of the key concepts in medical genetics like DNA, genes, and enzymes. Different methods for gene delivery are also summarized, including viral vectors, physical methods, and chemical methods. Applications of gene therapy for cancer, neurological disorders, and other diseases are briefly mentioned.
INTRODUCTION
DNA VACCINES
GENE THERAPY
TIME LINE OF DEVELOPING GENE THERAPY
GENE THERAPY STRATEGIES
TECHNOLOGY OF CLASSICAL GENE THERAPY
PRINCIPLES OF GENE TRANSFER
VECTORS
VIRAL VECTORS
NON-VIRAL VECTORS
APPLICATIONS OF GENE THERAPY
ETHICAL IMPLICATIONS
THE FUTURE
CONCLUSION
REFERENCES
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.
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.
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
Introduction
Approaches to Gene Therapy
Vectors in Gene Therapy
Non-viral Methods
Physical Methods for Improving DNA Transfer
Chemical Methods for Improving DNA Transfer
Advantages and Disadvantages of Gene Therapy
Applications of Gene Therapy
Challenges
The document discusses gene therapy as a promising approach for treating various diseases. It provides a brief history of gene therapy and describes some of the early clinical trials. It then explains some of the key concepts in medical genetics like DNA, genes, and enzymes. Different methods for gene delivery are also summarized, including viral vectors, physical methods, and chemical methods. Applications of gene therapy for cancer, neurological disorders, and other diseases are briefly mentioned.
INTRODUCTION
DNA VACCINES
GENE THERAPY
TIME LINE OF DEVELOPING GENE THERAPY
GENE THERAPY STRATEGIES
TECHNOLOGY OF CLASSICAL GENE THERAPY
PRINCIPLES OF GENE TRANSFER
VECTORS
VIRAL VECTORS
NON-VIRAL VECTORS
APPLICATIONS OF GENE THERAPY
ETHICAL IMPLICATIONS
THE FUTURE
CONCLUSION
REFERENCES
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.
This document provides an overview of gene therapy, including what it is, different types and approaches, vectors used, methods of delivery, advantages and disadvantages. Gene therapy involves inserting a normal gene to replace an abnormal gene responsible for a disease. It can be done via in vivo or ex vivo methods. Viral and non-viral vectors are used to deliver genes. While gene therapy holds promise to treat genetic diseases, it also faces challenges such as short-lived effects and safety issues.
DNA lipofection - Efficiency in invitro and invivo transfectionpugazhenthi6
This document discusses DNA lipofection and its efficiency in in vitro and in vivo transfection. It begins by introducing gene therapy and its goal of introducing genes to prevent or cure diseases. It then describes the two main types of gene therapy: somatic cell gene therapy, which does not affect future generations; and germ line gene therapy, which is heritable but not currently attempted. The document outlines approaches to gene therapy, including ex vivo and in vivo methods. It discusses various vectors used in gene therapy such as viral vectors like retroviruses and adenoviruses and non-viral vectors like lipoplexes. The document concludes by noting both the disadvantages of potential immune responses and difficulties treating multiple gene disorders through gene therapy and
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.
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.
The concept of transferring genes to tissues for clinical applications has been discussed for nearly half a century, but the ability to manipulate genetic material via recombinant DNA technology has brought this goal to reality. ‘Gene Therapy’ covers both the research and clinical applications of the new genetic therapy techniques currently being developed. The application of molecular biology has revolutionized researchers understanding of many diseases and has been readily applied for diagnostic purposes. Now-a-day this is originally conceived as a way to treat life-threatening disorders (inborn errors, cancers) refractory to conventional treatment, gene therapy now is considered for many non–life-threatening conditions, including those adversely affecting a patient’s quality of life. The lack of suitable treatment has become a rational basis for extending the scope of gene therapy. It is not very far, the justifiable optimism that with increased biotechnological improvement, gene therapy will become a standard part of clinical practice.
Gene transfer technologies can be used to treat diseases by inserting therapeutic genes into cells. There are viral and non-viral methods of gene transfer. Viral methods use viruses like retroviruses, adenoviruses, and adeno-associated viruses to efficiently deliver genes. Non-viral methods include mechanical techniques like electroporation, microinjection, and biolistics (gene gun), as well as chemical methods like liposomes, calcium phosphate, and polyethylene glycol. Each method has advantages and limitations for different applications in research and potential gene therapy.
Lectins are carbohydrate-binding proteins or glyco-proteins binding selectively without the involvement of enzymes, Gene responsible for expression lection found in chromosome 10q11.2-q21
Found in plnats grains, legume, soy bean, kidney bean
Lectins recognize tumor marker which play important role for diagnosing tumor cell, screening tumour and able to detect subtle neoplastic changes
NUCLEIC ACID BASED THERAPEUTIC DELIVERY SYSTEM by pramesh..pptxPRAMESHPANWAR1
Name of the title: Nucleic Acid-Based Therapeutic Delivery System.
It includes information about nucleic acid, gene therapy, and its type, a method to deliver the desired DNA, i.e., vectors and their types, with proper examples and diagrams, and how these things help in delivering a nucleic acid-based therapeutic drug delivery system.
The document discusses gene therapy and its potential to treat genetic diseases. It describes how gene therapy works by introducing functional genes into cells to replace defective genes causing disease. The first approved gene therapy treated a girl with ADA-SCID by inserting a functional ADA gene. While promising, gene therapy faces challenges like short-lived effects and safety issues that must still be addressed.
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 involves inserting normal genes into patients to replace abnormal genes that cause disease. It is being studied for many diseases like immunodeficiencies, hemophilia, Parkinson's, and cancer. The first gene therapy occurred in 1990 and involved treating a genetic immune deficiency. While it offers potential cures, there are also risks and ethical concerns around its use.
Gene therapy and stem cells are discussed. [1] Gene therapy involves delivering genetic material into cells for therapeutic purposes using vectors. It aims to replace defective genes, enhance gene expression, or suppress harmful genes. [2] Stem cells are undifferentiated cells that can develop into specialized cell types and have self-renewal abilities. There are embryonic, adult tissue specific, and induced pluripotent stem cells. Gene therapy and stem cells offer potential treatments for diseases like cancer, genetic disorders, and tissue regeneration. However, safety and delivery challenges remain.
Gene therapy types advantags disadvantagesSUJITHA MARY
Gene therapy involves inserting normal genes into patients to replace abnormal genes responsible for disease. There are two main types of gene therapy: ex vivo involves removing cells, modifying them, and reinserting them; in vivo delivers genes directly into target tissues. Viral and non-viral vectors are used to deliver genes. While gene therapy has potential to treat many diseases, it also has disadvantages like short-term effects and safety risks that must still be addressed.
Gene therapy aims to alter the phenotype of diseased cells by introducing exogenous nucleic acids like DNA. It involves choosing a cellular target, effector mechanism, delivery method, and effector gene. For cancer, common targets are tumor cells themselves or normal cells like stem cells. Effector mechanisms include gene replacement, suicide genes, and RNA-directed strategies. Delivery can be viral, non-viral, or involve artificial virus-like particles. Effector genes, promoters, and suicide genes must be chosen to match the intended target and effect. Some gene therapy strategies have shown effectiveness in clinical trials for cancers like breast and prostate.
Gene therapy and cell therapy hold promise for treating cardiovascular diseases. The document discusses gene therapy strategies using viral and non-viral vectors to target conditions like heart failure, atherosclerosis, and ischemia. Key molecular targets discussed for heart failure include SERCA2a, SDF-1, and genes involved in calcium handling. Challenges for cardiovascular gene therapy include developing efficient methods for delivering gene vectors to target tissues.
Gene therapy involves inserting normal genes into cells to treat genetic diseases. There are two main methods - using viruses as vectors to deliver genes (viral vectors) or delivering naked DNA. Researchers are studying gene therapy for diseases like cancer, HIV, hemophilia and more. Some successes include treating blindness caused by a genetic defect using a virus to deliver a healthy gene to the eyes. Gene therapy has also reduced Parkinson's disease symptoms by delivering a gene to the brain to increase inhibitory neurotransmitters. While offering hope, gene therapy also faces challenges in terms of safety, cost and ethical issues.
This document discusses components of a gene therapy strategy for cancer treatment. It describes introducing exogenous DNA into cancer cells to alter gene expression and phenotype. There are two main aims - replacing defective tumor suppressor genes or introducing genes to kill cancer cells. Delivery methods include viral vectors, physical methods like injection, and chemical methods using liposomes. The document also discusses targeting tumor cells or normal cells like stem cells, using suicide genes or gene replacement, and overcoming challenges of systemic delivery.
Definition, Gene therapy, types of gene therapy, germline gene therapy, somatic cell gene therapy, basic process of gene therapy and potential targets for gene therapy.
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
This document provides an overview of gene therapy, including what it is, different types and approaches, vectors used, methods of delivery, advantages and disadvantages. Gene therapy involves inserting a normal gene to replace an abnormal gene responsible for a disease. It can be done via in vivo or ex vivo methods. Viral and non-viral vectors are used to deliver genes. While gene therapy holds promise to treat genetic diseases, it also faces challenges such as short-lived effects and safety issues.
DNA lipofection - Efficiency in invitro and invivo transfectionpugazhenthi6
This document discusses DNA lipofection and its efficiency in in vitro and in vivo transfection. It begins by introducing gene therapy and its goal of introducing genes to prevent or cure diseases. It then describes the two main types of gene therapy: somatic cell gene therapy, which does not affect future generations; and germ line gene therapy, which is heritable but not currently attempted. The document outlines approaches to gene therapy, including ex vivo and in vivo methods. It discusses various vectors used in gene therapy such as viral vectors like retroviruses and adenoviruses and non-viral vectors like lipoplexes. The document concludes by noting both the disadvantages of potential immune responses and difficulties treating multiple gene disorders through gene therapy and
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.
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.
The concept of transferring genes to tissues for clinical applications has been discussed for nearly half a century, but the ability to manipulate genetic material via recombinant DNA technology has brought this goal to reality. ‘Gene Therapy’ covers both the research and clinical applications of the new genetic therapy techniques currently being developed. The application of molecular biology has revolutionized researchers understanding of many diseases and has been readily applied for diagnostic purposes. Now-a-day this is originally conceived as a way to treat life-threatening disorders (inborn errors, cancers) refractory to conventional treatment, gene therapy now is considered for many non–life-threatening conditions, including those adversely affecting a patient’s quality of life. The lack of suitable treatment has become a rational basis for extending the scope of gene therapy. It is not very far, the justifiable optimism that with increased biotechnological improvement, gene therapy will become a standard part of clinical practice.
Gene transfer technologies can be used to treat diseases by inserting therapeutic genes into cells. There are viral and non-viral methods of gene transfer. Viral methods use viruses like retroviruses, adenoviruses, and adeno-associated viruses to efficiently deliver genes. Non-viral methods include mechanical techniques like electroporation, microinjection, and biolistics (gene gun), as well as chemical methods like liposomes, calcium phosphate, and polyethylene glycol. Each method has advantages and limitations for different applications in research and potential gene therapy.
Lectins are carbohydrate-binding proteins or glyco-proteins binding selectively without the involvement of enzymes, Gene responsible for expression lection found in chromosome 10q11.2-q21
Found in plnats grains, legume, soy bean, kidney bean
Lectins recognize tumor marker which play important role for diagnosing tumor cell, screening tumour and able to detect subtle neoplastic changes
NUCLEIC ACID BASED THERAPEUTIC DELIVERY SYSTEM by pramesh..pptxPRAMESHPANWAR1
Name of the title: Nucleic Acid-Based Therapeutic Delivery System.
It includes information about nucleic acid, gene therapy, and its type, a method to deliver the desired DNA, i.e., vectors and their types, with proper examples and diagrams, and how these things help in delivering a nucleic acid-based therapeutic drug delivery system.
The document discusses gene therapy and its potential to treat genetic diseases. It describes how gene therapy works by introducing functional genes into cells to replace defective genes causing disease. The first approved gene therapy treated a girl with ADA-SCID by inserting a functional ADA gene. While promising, gene therapy faces challenges like short-lived effects and safety issues that must still be addressed.
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 involves inserting normal genes into patients to replace abnormal genes that cause disease. It is being studied for many diseases like immunodeficiencies, hemophilia, Parkinson's, and cancer. The first gene therapy occurred in 1990 and involved treating a genetic immune deficiency. While it offers potential cures, there are also risks and ethical concerns around its use.
Gene therapy and stem cells are discussed. [1] Gene therapy involves delivering genetic material into cells for therapeutic purposes using vectors. It aims to replace defective genes, enhance gene expression, or suppress harmful genes. [2] Stem cells are undifferentiated cells that can develop into specialized cell types and have self-renewal abilities. There are embryonic, adult tissue specific, and induced pluripotent stem cells. Gene therapy and stem cells offer potential treatments for diseases like cancer, genetic disorders, and tissue regeneration. However, safety and delivery challenges remain.
Gene therapy types advantags disadvantagesSUJITHA MARY
Gene therapy involves inserting normal genes into patients to replace abnormal genes responsible for disease. There are two main types of gene therapy: ex vivo involves removing cells, modifying them, and reinserting them; in vivo delivers genes directly into target tissues. Viral and non-viral vectors are used to deliver genes. While gene therapy has potential to treat many diseases, it also has disadvantages like short-term effects and safety risks that must still be addressed.
Gene therapy aims to alter the phenotype of diseased cells by introducing exogenous nucleic acids like DNA. It involves choosing a cellular target, effector mechanism, delivery method, and effector gene. For cancer, common targets are tumor cells themselves or normal cells like stem cells. Effector mechanisms include gene replacement, suicide genes, and RNA-directed strategies. Delivery can be viral, non-viral, or involve artificial virus-like particles. Effector genes, promoters, and suicide genes must be chosen to match the intended target and effect. Some gene therapy strategies have shown effectiveness in clinical trials for cancers like breast and prostate.
Gene therapy and cell therapy hold promise for treating cardiovascular diseases. The document discusses gene therapy strategies using viral and non-viral vectors to target conditions like heart failure, atherosclerosis, and ischemia. Key molecular targets discussed for heart failure include SERCA2a, SDF-1, and genes involved in calcium handling. Challenges for cardiovascular gene therapy include developing efficient methods for delivering gene vectors to target tissues.
Gene therapy involves inserting normal genes into cells to treat genetic diseases. There are two main methods - using viruses as vectors to deliver genes (viral vectors) or delivering naked DNA. Researchers are studying gene therapy for diseases like cancer, HIV, hemophilia and more. Some successes include treating blindness caused by a genetic defect using a virus to deliver a healthy gene to the eyes. Gene therapy has also reduced Parkinson's disease symptoms by delivering a gene to the brain to increase inhibitory neurotransmitters. While offering hope, gene therapy also faces challenges in terms of safety, cost and ethical issues.
This document discusses components of a gene therapy strategy for cancer treatment. It describes introducing exogenous DNA into cancer cells to alter gene expression and phenotype. There are two main aims - replacing defective tumor suppressor genes or introducing genes to kill cancer cells. Delivery methods include viral vectors, physical methods like injection, and chemical methods using liposomes. The document also discusses targeting tumor cells or normal cells like stem cells, using suicide genes or gene replacement, and overcoming challenges of systemic delivery.
Definition, Gene therapy, types of gene therapy, germline gene therapy, somatic cell gene therapy, basic process of gene therapy and potential targets for gene therapy.
Similar to VIRAL AND NON VIRAL GENE TRANSFER.pptx (20)
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
Integrating Ayurveda into Parkinson’s Management: A Holistic ApproachAyurveda ForAll
Explore the benefits of combining Ayurveda with conventional Parkinson's treatments. Learn how a holistic approach can manage symptoms, enhance well-being, and balance body energies. Discover the steps to safely integrate Ayurvedic practices into your Parkinson’s care plan, including expert guidance on diet, herbal remedies, and lifestyle modifications.
- Video recording of this lecture in English language: https://youtu.be/kqbnxVAZs-0
- Video recording of this lecture in Arabic language: https://youtu.be/SINlygW1Mpc
- Link to download the book free: https://nephrotube.blogspot.com/p/nephrotube-nephrology-books.html
- Link to NephroTube website: www.NephroTube.com
- Link to NephroTube social media accounts: https://nephrotube.blogspot.com/p/join-nephrotube-on-social-media.html
share - Lions, tigers, AI and health misinformation, oh my!.pptxTina Purnat
• Pitfalls and pivots needed to use AI effectively in public health
• Evidence-based strategies to address health misinformation effectively
• Building trust with communities online and offline
• Equipping health professionals to address questions, concerns and health misinformation
• Assessing risk and mitigating harm from adverse health narratives in communities, health workforce and health system
Cell Therapy Expansion and Challenges in Autoimmune DiseaseHealth Advances
There is increasing confidence that cell therapies will soon play a role in the treatment of autoimmune disorders, but the extent of this impact remains to be seen. Early readouts on autologous CAR-Ts in lupus are encouraging, but manufacturing and cost limitations are likely to restrict access to highly refractory patients. Allogeneic CAR-Ts have the potential to broaden access to earlier lines of treatment due to their inherent cost benefits, however they will need to demonstrate comparable or improved efficacy to established modalities.
In addition to infrastructure and capacity constraints, CAR-Ts face a very different risk-benefit dynamic in autoimmune compared to oncology, highlighting the need for tolerable therapies with low adverse event risk. CAR-NK and Treg-based therapies are also being developed in certain autoimmune disorders and may demonstrate favorable safety profiles. Several novel non-cell therapies such as bispecific antibodies, nanobodies, and RNAi drugs, may also offer future alternative competitive solutions with variable value propositions.
Widespread adoption of cell therapies will not only require strong efficacy and safety data, but also adapted pricing and access strategies. At oncology-based price points, CAR-Ts are unlikely to achieve broad market access in autoimmune disorders, with eligible patient populations that are potentially orders of magnitude greater than the number of currently addressable cancer patients. Developers have made strides towards reducing cell therapy COGS while improving manufacturing efficiency, but payors will inevitably restrict access until more sustainable pricing is achieved.
Despite these headwinds, industry leaders and investors remain confident that cell therapies are poised to address significant unmet need in patients suffering from autoimmune disorders. However, the extent of this impact on the treatment landscape remains to be seen, as the industry rapidly approaches an inflection point.
Clinic ^%[+27633867063*Abortion Pills For Sale In Tembisa Central19various
Clinic ^%[+27633867063*Abortion Pills For Sale In Tembisa Central Clinic ^%[+27633867063*Abortion Pills For Sale In Tembisa CentralClinic ^%[+27633867063*Abortion Pills For Sale In Tembisa CentralClinic ^%[+27633867063*Abortion Pills For Sale In Tembisa CentralClinic ^%[+27633867063*Abortion Pills For Sale In Tembisa Central
Promoting Wellbeing - Applied Social Psychology - Psychology SuperNotesPsychoTech Services
A proprietary approach developed by bringing together the best of learning theories from Psychology, design principles from the world of visualization, and pedagogical methods from over a decade of training experience, that enables you to: Learn better, faster!
TEST BANK For Basic and Clinical Pharmacology, 14th Edition by Bertram G. Kat...rightmanforbloodline
TEST BANK For Basic and Clinical Pharmacology, 14th Edition by Bertram G. Katzung, Verified Chapters 1 - 66, Complete Newest Version.
TEST BANK For Basic and Clinical Pharmacology, 14th Edition by Bertram G. Katzung, Verified Chapters 1 - 66, Complete Newest Version.
TEST BANK For Basic and Clinical Pharmacology, 14th Edition by Bertram G. Katzung, Verified Chapters 1 - 66, Complete Newest Version.
TEST BANK For Basic and Clinical Pharmacology, 14th Edition by Bertram G. Katzung, Verified Chapters 1 - 66, Complete Newest Version.
Histololgy of Female Reproductive System.pptxAyeshaZaid1
Dive into an in-depth exploration of the histological structure of female reproductive system with this comprehensive lecture. Presented by Dr. Ayesha Irfan, Assistant Professor of Anatomy, this presentation covers the Gross anatomy and functional histology of the female reproductive organs. Ideal for students, educators, and anyone interested in medical science, this lecture provides clear explanations, detailed diagrams, and valuable insights into female reproductive system. Enhance your knowledge and understanding of this essential aspect of human biology.
Post-Menstrual Smell- When to Suspect Vaginitis.pptx
VIRAL AND NON VIRAL GENE TRANSFER.pptx
1. PRESENTED BY :-
TRISHA KAR
M.PHARM ( 2nd SEMESTER)
UNDER GUIDANCE BY Dr. AMITAVA ROY
DEPARTMENT OF PHARMACEUTICAL TECHNOLOGY
UNIVERSITY OF NORTH BENGAL
VIRALAND NON VIRAL GENE TRANSFER
2. GENE THERAPY :-
Gene therapy is the process of introducing foreign genomic materials into host cells to elicit a
therapeutic benefit.
It can be also defined as an experimental technique for correcting defective genes that are
responsible for disease development.
The most common form of gene therapy involves inserting a normal gene to replace an
abnormal gene.
The first approved gene therapy experiment occurred on September 14,1990 in US, when
Ashanti De Silva was treated for ADA-SCID.
In the future, this technique may allow doctors to treat a disorder by inserting a gene into a
patient's cells instead of using drugs or surgery[1].
3. It defined as a technique to efficiently and stably introduce foreign material into
the genome of the target cells.
The insertion of unrelated, therapeutic genetic information in the form of DNA
into target cells[2].
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.
It provides a novel approach for the investigation and potential treatment of a
variety of disease[2].
GENE TRANSFER
4. NON VIRAL METHOD
VIRAL METHOD
BASED ON THE VECTOR THAT ARE USED IN GENE TRANSFER TECHNIQUES
CAN BE DIVIDED AS
GENE TRANSFER
TECNIQUES
5. NON VIRAL
METHOD
Non viral systems comprise all the physical and chemical systems
Generally include either chemical methods, such as cationic liposomes and polymers,
or physical methods, such as gene gun, electroporation, particle bombardment,
ultrasound utilization, and magnetofection.
Efficiency of this system is less than viral systems in gene transduction, but their
cost-effectiveness, availability, and no limitation in size of transgenic DNA compared
with viral system[3].
6. PHYSICAL
METHOD
1. DNA particle bombardment by Gene gun
• It is an ideal alternative technique to injection of naked
DNA.
• Gold or tungsten spherical particles (1–3 μm diameter)
are coated with plasmid DNA and then accelerated to
high speed by pressurized gas to penetrate into target
tissue cells.
• Actually it is a modification of a technique called
“biolistic,” originally developed for plant transgenesis,
but now used for in vitro and in vivo gene delivery into
mammalian cells too[4]. FIG 1: GENE GUN[4]
7. 2.ULTRASOUND
• Ultrasound can make some nanomeric pores in membrane to facilitate intracellular
delivery of DNA particles into cells of internal organs or tumors
• The most important limitation of the system is low efficiency of it, especially invivo[5].
3. ELECTROPORATION
• Electroporation is temporary destabilization of the cell membrane targeted tissue by
insertion of a pair of electrodes into it.
• DNA molecules in the surrounding media of the destabilized membrane would be able
to penetrate into cytoplasm and nucleoplasm of the cell
• Electroporation has been used in vivo for many types of tissues, such as skin, muscle,
lung, HPRT gene delivery, and tumor treatment[6].
• Some problems in this method are :-
The difficulty in surgical procedure in the placement of electrodes into the internal tissues
The high voltage applied to tissue might damage the organ and affect genomic DNA
stability[6]
8. MAGNETOFECTION
• In this method the magnetic fields are used to concentrate particles containing nucleic acid
into the target cells.
• In this way, the magnetic force allows a very rapid concentration of the entire applied
vector dose onto cells, so that 100% of the cells get in contact with a significant vector
dose.
• This technique is non invasive and can precisely target genetic material to the desired site
while increasing the gene expression
• Magnetofection has been adapted to all types of nucleic acids ,nonviral transfection
systems and viruses. It has been successfully tested on a broad range of cell lines,
hard-to-transfect and primary cells[7].
FIG 2 :- PROCESS OF MAGNETOFECTION[7].
9. CHEMICAL
METHOD
1.CATIONIC LIPOSOME
• Cationic liposomes are the more important current nonviral polycationic systems, which
compact negatively charged nucleic acids lead to the formation of nanomeric complexes.
• Cationic liposomes are being used in gene delivery into lung, skeletal muscles, spleen,
kidney, liver, testis, heart and skin cells[8].
• Cationic liposomes have unique characteristics such as[8] :-
Capability to incorporate hydrophilic and hydrophobic drugs
Low toxicity
No activation of immune system
Targeted delivery of bioactive compounds to the site of action
10. 2. CATIONIC POLYMERS
• Cationic polymers have also been used extensively for gene transfer.
• Upon mixing with DNA, these polymers form nanosized complexes, often called
polyplexes.
• Among cationic polymers, PEI is considered one of the most effective polymer-
based transfection agents.
• PEI leads to an influx of chloride counter ions within the compartment and a build
up of osmotic pressure that causes the swelling and rupture of the endosomal
membrane.
• Recently, more polymers with improved biocompatibility and biodegradability have
been reported demonstrating equal or superior performance comparing to
nondegradable PEIS[9].
11. Viral
method
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.
There are a wide variety of vectors used to deliver DNA or oligo nucleotides
into mammalian cells, either in vitro or in vivo.
The most common vector system based on retroviruses, adenoviruses, herpes
simplex viruses, adeno associated viruses[9].
12. THREE COMMONLY USED VIRAL GENE
TRANSFER SYSTEMS ARE [9] :-
RETRO VIRUS (RV) Adeno virus(AV) Adeno Associated
Virus(AAV)
13. 1. RETROVIRUS VECTOR
• 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.
• Retroviruses are useful for ex vivo delivery of somatic cells because of their ability to
linearly integrate into host cell genome[10].
A P P L I C A T I O N
Treatment of T-lymphocyte deficiency(ADA), Tumour Infiltrating Lymphocytes(TIL),
Bone marrow cells(ADA deficiency, Gauchers disease), hepatocytes (LDL receptor
deficiency) and melanoma.
In-vivo gene transfer using retro viral vectors for suicide genes used in brain tumour.
Retroviral vectors also have been applied for familial hyperlipidemia gene therapy
and tumor vaccination[10].
14. 2.ADENO VIRUS VECTOR
• 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.
• Sterotype2 and type 5 can be utilized for transferring both dividing and non dividing cells
and have low host specificity.
• Natural and acute immunologic responses against adenoviruses have made their clinical
application limited to a few tissues, such as liver, lung or localized cancer gene therapy[11].
In 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[11].
APPLICATION
15. 3. ADENO-ASSOCIATED VIRUS VECTOR
• Adeno-associated vectors (AAV) are like adenoviral vectors in their features but because
of having some deficiency in their replication and pathogenicity, are safer than adenoviral
vectors.
• Members of Parvo virus family.
• Heat stable and resistant to various chemicals
• Depend on virus - cannot replicate its own, another virus is necessary for replicate.
• Another special character of AAV is their ability to integrate into a specific site on
chromosome 19 with no noticeable effects cause long-term expression in vivo[12].
A P P L I C A T I O N
Major disadvantages of these vectors are complicated process of vector production and the
limited transgene capacity of the particles.
Used in haematopoietic stem cells for treatment of B- thalassemia and sickle cell anaemia.
B-thalassemia erythrocyte contains insufficient ẞ- globin chain whereas, mutant B- globin
chains are produced in sickle cell[12].
16. CONCLUSIO
N
Although numerous viral and nonviral gene delivery systems have been developed in the last
3 decades, all of them have some disadvantages that have made some limitations in their
clinical application and yet no delivery system has been designed that can be applied in gene
therapy of all kinds of cell types in vitro and in vivo with no limitation and side effects.
However, some delivery systems has been explored, which can be efficient for gene delivery
to specific cells or tissues. So it seems that the process of developing successful delivery
systems, especially nonviral systems, for use in in vivo. Totally, key steps effective in
improving the currently available systems include the following:
Improving extracellular targeting and delivery,
Enhancing intracellular delivery and long-time
17. REFERENCE
1. McDonough PG. The ethics of somatic and germline gene therapy. Ann N YAcad Sci 1997;816:378-82
2. Audouny SA, Deleij LF, Hoekstra D, Molema G. In vivo characteristics of cationic liposomes as delivery vectors for gene therapy. Pharm Res
2002;19:1599-6005.
3. Varga CM, Hong K, Lanf Furburger DA. Quantitative analysis of synthesis gene delivery vector design properties. Mol Ther 2001;4:438-46.
4. Wolff JA, Ludtke JJ, Acsadi G, Williams P, Jani A. Long term persistence of plasmid DNA and foreign gene expression in mouse muscle. Hum
Mol Genet 1992;1:363-9.
5. Jiang J, Yamato E, Miyazaki J. Intravenous delivery of naked plasmid DNA for in vivo cytokine expression. Biochem Biophys Res Commun
2001;289:1088-92.
6. Dean DA, Machado Aranda D, Blair Parks K, Yeldandi AV, Young JL. Electroporation as a method for high level nonviral gene transfer to the
lung. Gene Ther 2003;10:1608-15
7. Plank C, Schillinger U, Scherer F, Bergemann C, Rémy JS, Krötz F, et al. The magnetofection method: Using magnetic force to enhance gene
delivery. Biol Chem 2003;384:737-47.
8. Schnyder A, Huwyler J. Drug transport to brain with targeted liposomes. NeuroRx 2005;2:99-107
9. Tang MX, Szoka FC. The influence of polymer structure on the interaction of cationic polymers with DNA and morphology of the resulting
complexes. Gene Ther 1997;4:823-32
10. Hacein-Bey-Abina S, Von Kalle C, Schmidt M, McCormack MP, Wulffraat N, Leboulch P, et al. LMO2-associated clonal T cell proliferation in
two patients after gene therapy for SCID-XI. Science 2003;302:415-9.
11. Marshall E. Gene therapy death prompts review of adenovirus vector. Science 1999;286:2244-5.
12. Kay MA, Manno CS, Ragni MV, Larson PJ, Couto LB, McClelland A, et al. Evidence for gene transfer and expression of factor IX in
haemophilia B patients treated with an AAV vector. Nat Genet 2000;24:257-61.