Gene Therapy Ppt

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

Introduction Various approaches Types of gene delivery Gene therapy of various diseases conclusion

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.

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.

Gene modification Gene transfer Gene transfer in specific cell line

Replacement therapy

Corrective gene therapy

Physical Chemical Biological

Somatic

Germ line

Retroviruses Adenoviruses Adeno-associated viruses Herpes Simplex Virus Alpha virus

Gene gun Liposome mediated gene delivery Polymer based gene delivery Electroporation Proton sponge

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.

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.

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 fibrosis subjects receiving pulmonary delivery of GL-67 proportions 1:2:0.05) complexed to 7.9–21.1 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.

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.

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

Vectors or carriers of therapeutic gene cause problems.

Expensive therapy

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..

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

www.genetherapy.net www.nature.com www.genomics.energy.gov