Gene Therapy Ppt 4e5s1v

m By Jyothi kiranmai .G (M.Pharm Pharmacology) MALLA REDDY COLLEGE OF PHARMACY

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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.

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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.

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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.

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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.

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Therapeutic gene added to the cell is functionally active for only short span time.

Vectors or carriers of therapeutic gene cause problems.

Expensive therapy

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R Pharmaceutical biotechnology S.P.Vyas and V.K. Dixit.

www.genetherapy.net

www.nature.com

www.genomics.energy.gov