Gene therapy is a new tool used in combating different diseases. The majority of gene therapy clinical trials are focused on cancer and so it was no coincidence that the first commercial treatment in 2003 was for neoplasia. Currently there are a wide
Gene therapy is a contemporary therapeutic intervention with recent positive results and regulatory approvals either completed or expected in the next several years for various conditions. The evolving view is that gene therapy will ultimately offer
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Pastor Gene Lingerfelt is the founder and senior pastor. As a pastor and author, Pastor Gene has a reputation for his practical and relevant teaching. His books include "The God Touch" and "10 Words That Can Change Your Life.
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Descripción: Study Guide of the selfish gene
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Study Guide of the selfish gene
GENE THERAPY
By Jyothi kiranmai .G (M.Pharm Pharmacology) MALLA REDDY COLLEGE OF PHARMACY
CONTENTS ´
Introduction
´
Various approaches
´
Types of gene delivery
´
Gene therapy of various diseases
´
conclusion
INTRODUCTION
Gene ?? ´
According to the official Guidelines for Human Gene Nomenclature, a gene is defined as "a DNA segment that contributes to phenotype/function.
1969 - The first single gene is isolated. 1970 - The first artificial gene is made.
GENE THERAPY
Definition:
Gene delivery, the insertion of genes (e.g. via retroviral vectors) into selected cells in the body in order to: - Cause those cells to produce specific therapeutic agents. - Cause those cells to become (more) susceptible to a conventional therapeutic agent that previously was ineffective against that particular condition/disease. - Cause those cells to become less susceptible to a conventional therapeutic agent. - Counter the effects of abnormal (damaged) tumour suppressor genes via insertion of normal tumour suppressor genes.
- Introduce other therapeutics into cells.
APPROACHES FOR GENE THERAPY ´
Gene modification
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Gene transfer
´
Gene transfer in specific cell line
GENE MODIFICATION ´
R eplacement
therapy
´
Corrective gene therapy
GENE TRANSFER ´
Physical
´
Chemical
´
Biological
GENE TRANSFER IN SPECIFIC CELL LINES ´
Somatic
´
Germ line
TYPES OF GENE DELIVERY
VIRAL MEDIATED ´
R etroviruses
´
Adenoviruses
´
Adeno-associated viruses
´
Herpes Simplex Virus
´
Alpha virus
ADENO VIRUS
NON VIRAL MEDIATED ´
Gene gun
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Liposome mediated gene delivery
´
Polymer
´
Electroporation
´
Proton
based gene delivery
sponge
GENE GUN
ELECTROPORATION
LIPOSOMES A mixture of polycataionic lipid and neutral lipid will result in formation of unilamellar liposome vesicles that have a net positive charge due to positive amine groups on these molecules.
PROTON SPONGE
FIRST GENE THERAPY ´
ADA: The First Gene Therapy Trial
´
A four-year old girl became the first gene therapy patient on September 14, 1990 at the NIH Clinical Center. She has adenosine deaminase (ADA) deficiency, a genetic disease which leaves her defenseless against infections. WHITE BLOOD CELLS were taken from her, and the normal genes for making adenosine deaminase were inserted into them. The corrected cells were reinjected into her. Dr. W. French Anderson helped develop this landmark clinical trial when he worked at the National Heart, Lung, and Blood Institute.
´
1985
´
The laboratories of Drs. W. French Anderson and Michael Blaese in the National Heart, Lung, and Blood Institute and the National Cancer Institute worked together to show that cells from patients with ADA deficiency can be corrected in tissue culture . They used a retrovirusto carry the correct human ADA gene to the cells. Here, Drs. Kenneth Culver (then of the NHLBI), W. French Anderson, and Michael Blaese pose with gene therapy patients.
APPLICATIONS ´ ´
´
´ ´
Gene therapy for insulin dependent diabetis melitus Gene therapy for hemophilia B novel self complementary AAV vector encoding a codon-optimised human F IX transgene (scAAV2/8-LP1-hFIXco) into adult subjects with severe HB Gene therapy for cystic fibrosis The most recently reported trial involved eight cystic Àbrosis subjects receiving pulmonary delivery of GL-67 proportions 1:2:0.05) complexed to 7.9²21.1 mg of CFTR plasmid DNA [88]. Gene therapy for cardiovascular diseases. Gene therapy for nervous system disorders.
´
Results of world's first gene therapy for inherited blindness show sight improvement. 28 April 2008. UK researchers from the UCL Institute of Ophthalmology and Moorfields Eye Hospital NIHR Biomedical Research Centre have announced results from the world·s first clinical trial to test a revolutionary gene therapy treatment for a type of inherited blindness. The results, published today in the New England Journal of Medicine, show that the experimental treatment is safe and can improve sight. The findings are a landmark for gene therapy technology and could have a significant impact on future treatments for eye disease.
RECENT DEVELOPMENTS IN GENE THERAPY ´
Genes carried in nanoparticles are used to treat cancer in mice
´
Gene therapy cured inheritance blindness in humans.
´
Gene therapy was successful in treating adenosine deaminase deficiency an auto immune dis.order in humans.
LIMITATIONS OF GENE THERAPY ´
Therapeutic gene added to the cell is functionally active for only short span time.
´
Vectors or carriers of therapeutic gene cause problems.
´
Expensive therapy
CONCLUSION ´
Almost every therapy which intended were succeeded in cell and animal models.
´
Its mark in treating humans is most awaiting one as all are in clinical trials«
´
Lets hope for a better way for treating diseases with ethical values..
REFERENCES ´
Pharmaceutical biotechnology S.P.Vyas and V.K. Dixit.