Plasmid technology the vector method pp
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Plasmid technology uses restriction enzymes to cut viral DNA and the DNA to be inserted, fusing them together. Virulence genes are removed from viruses which are then replicated and injected, allowing the new gene to be expressed in cells and passed on through replication. Researchers have developed a viral vector delivering a gene that could cure Choroideremia with a single injection, though unpredictability of DNA insertion and using disabled viruses raise some ethical concerns. This technique aims to cure more genetic diseases in the future.
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A gene is region of DNA that encodes function and chromosome consist of long DNA strands containing many genes.
A human chromosome can have up to 500 million base pair of DNA with thousands of genes.
Technique responsible for correcting the defective genes responsible for disease development is called Gene Therapy
DNA lipofection - Efficiency in invitro and invivo transfection
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.
Gene therapy involves inserting normal genes into individuals to replace defective genes that cause disease. It has been used to treat various genetic diseases and cancers since the 1990s. While it offers promise for permanent treatment, gene therapy still faces challenges like short-term effects, immune responses, high costs, and difficulties with gene delivery methods that have limited its effectiveness so far. Continued research aims to overcome these obstacles.
Gene delivery and gene therapy . Gene delivery , Gene therapy .
A good comprehensive review of gene delivery and gene therapy. especially for master of pharmacy 2nd-semester students as per the PCI syllabus of subject Molecular pharmaceutics.
List of contents under this ppt :
{A} GENE THERAPY
(1) Definition
(2) Introduction
(3) History
(4) Ex-Vivo gene therapy
(5) In-Vivo gene therapy
(6) Germline gene therapy
(7) Advantages of gene therapy
(8) Disadvantages of gene therapy
(9) Potential target diseases for gene therapy
a. inherited disorders :- ADA SCID, Chronic granulomatous, Hemophelia
b. Cancer
{B} GENE DELIVERY
(1) Definition
(2) Introduction
(3) Types of vectors
a. Viral :- Retrovirus, Adenovirus, Adeno associated virus, Herps simplex virus
b. Non viral :-
Physical methods - Gene gun, Microinjection, Electroporation, Sonoporation
Chemical methods - Oligonucleotides, Lipoplexes, Polyplexes, Dendrimers, Nanoparticles.
1. gene therapy
Gene therapy involves inserting normal genes into a person's cells to treat a disease. There are several strategies for gene therapy, including ex vivo and in vivo methods. Viral vectors are commonly used to deliver therapeutic genes to target cells. However, gene therapy has faced challenges such as short-lived effects, immune responses, and safety issues with viral vectors. Continued research is working to overcome these issues and further develop promising applications of gene therapy.
Gene Expression System-viral gene delivery Mpharm(Pharamaceutics)
Gene therapy can be broadly defined as the transfer of genetic material to cure a disease or at least to improve the clinical status of a patient.
One of the basic concepts of gene therapy is to transform viruses into genetic shuttles, which will deliver the gene of interest into the target cells.
Safe methods have been devised to do this, using several viral and non-viral vectors.
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.
The biggest hurdle faced by medical research in gene therapy is the availability of effective gene-carrying vectors that meet all of the following criteria:
Protection of transgene or genetic cargo from degradative action of systemic and endonucleases,
Delivery of genetic material to the target site, i.e., either cell cytoplasm or nucleus,
Low potential of triggering unwanted immune responses or genotoxicity,
Economical and feasible availability for patients .
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.
Lectins in 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
GENE THERAPY
This document provides an overview of gene therapy. It defines gene therapy as an experimental technique for correcting defective genes responsible for disease. It describes the main approaches like somatic cell gene therapy and germline gene therapy. It also discusses viral and non-viral vectors, delivery methods like in vivo and ex vivo, advantages like curing genetic diseases, and challenges like short-term effects and safety issues. Recent developments show promise for treating diseases like blindness and Parkinson's.
Group3_Team-Erythropoietin.pdf_ GENE THERAPY
Gene therapy is a medical approach that treats or prevents disease by correcting underlying genetic problems. It works by introducing genetic material into cells using viral or non-viral vectors. While first speculated in the 1960s, gene therapy has made progress in the past few decades, especially for single gene disorders. Current research focuses on developing gene therapy applications for cancers, infections like HIV, and other inherited disorders.
Dev gene therapy
The document discusses gene therapy, including:
1. Defining genes and discussing early milestones in gene isolation and engineering.
2. Explaining the goal of gene therapy to introduce normal genes to compensate for defective genes.
3. Describing various methods of gene delivery including viral and non-viral vectors.
4. Discussing challenges in targeting specific cells/tissues and ensuring safe expression levels.
Viral and non viral gene transfer
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.
Gene Therapy by Anupam Dwivedi (MS)
The document discusses genes and genetic disorders. It defines a gene as the basic physical and functional unit of heredity, and notes that genes are segments of DNA that code for specific traits. Genetic disorders are caused by variations or mutations in genes. The document then discusses recent discoveries in epigenetics, gene marking, and gene therapy. Gene therapy involves introducing new genes into cells to treat diseases, either by replacing defective genes, inactivating abnormal genes, or introducing new genes to fight diseases. Various gene therapy approaches and vectors, including viral vectors like retroviruses and adenoviruses, are described.
Similar to Plasmid technology the vector method pp (20)
DNA lipofection - Efficiency in invitro and invivo transfection
DNA lipofection - Efficiency in invitro and invivo transfection
Gene delivery and gene therapy . Gene delivery , Gene therapy .
Gene delivery and gene therapy . Gene delivery , Gene therapy .
Gene Therapy : information of gene therapy techniques
Gene Therapy : information of gene therapy techniques
Gene Expression System-viral gene delivery Mpharm(Pharamaceutics)
Gene Expression System-viral gene delivery Mpharm(Pharamaceutics)
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