Introduction to gene therapy

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Hello Dear all, In this first blog, I am bringing a brief introduction on the other line of treatment, the gene therapy, that some have considered as ''the medicine of the XXI century'' (1) but which is still being a focus of controversy, as far as its rationality is concerned. Concept of gene therapy: The DNA (Deoxyribonucleic acid) has been proved to hold the blueprint of life. The molecule guides the construction and development of living organisms. Genes, that are portions of DNA, are the life instructions of the cells; they tell the cells how to make proteins which take important part in the regulation, through various pathways, of many functions in our bodies. Then, it can easily be understood that any dysfunction in a given pathway, due to a defective gene would manifest itself as a disease. Therefore, it appears important to pay attention on genetic disorders. On the other side, the DNA has been defined as a ''transformative textbook of medicine, with insights that will give health providers immense new powers to treat, prevent and cure diseases'' (2). Gene therapy involves the transfer, alteration or removal of genetic material into or from cells to correct a cellular dysfunction or to provide new cellular function (3). Some History: The concept of gene therapy arose in the early 1970s as ''gene surgery'', originaly to treat life-threatening disorders (inborn errors, cancers) refractory to conventionally treatment. The growth of this technique was triggered in one hand with advancement in genetics that favored the ability to identify genetic abnormalities responsible, and on the other hand by the development on recombinant DNA which contributed to demonstrate the effectiveness of foreign genes in correcting defects and diseases in mammalian cells (4). First attempts of gene therapy in human are reported for Dr Anderson and his team working, in 1990, on a four-year-old girl with a Severe Combined Immunodefiency (SCID). Anderson and team used a retrovirus to insert gene into the T-lymphocytes of their patient (5). How it works? The factor in gene therapy treatment remains the pathogenic-free and efficient transfer of the gene into the target tissue: Gene therapy can be classified into two types: somatic gene therapy and germline gene therapy (6) i) Somatic gene therapy, which involves genetic alteration of the somatic cells and its replacement. This approach makes use of nonviral and viral vectors as delivery systems. ii) Germ line therapy involves modifying the gens in eggs or sperm cells by introduction of functional gens. Gene therapy, pros and cons: Beside its therapeutic potential, human gene therapy has raised many concerns in the scientific, ethical and religious domains (7) Ref 1- Verma I. M. and Weitzaman M.D. (2005) Gene therapy: twenty-first century medicine. Annual Review of Biochemistry 74 pp 711-738 2- Collins F. S. (2001) Contemplating the end of the beginning. Genome Research 11: 641-643 3- Culver K.W. (1994) Gene therapy A handbook for physians Mary Ann Liebert, Inc., New York 117pages 4- Friedman T. (1992) A brief history of gene therapy. Nature Genetics Doi: 10.1038/ng1092-93 5- Anderson F. W. Prospects for human gene therapy 6- 7-

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DIMEKO's picture
Author: DIMEKO


K Rajakrishna's picture

Dear demiko sir. Its nice to see your first scientific blog. Its short and precise. Can you elaborate on how the first gene for gene therapy was administered and what are the current ways to introduce it in the humans. For diagnosis and treatment.
DIMEKO's picture

Thank you Sir, I will try to go further in my next blogs Regards Didier Mekoo

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Sirisha Pingali's picture

Very nice sir. Does germ line therapy carry any limitations?? Are there any chances of mutations through this method?

Sirisha Pingali

Viswanadha Institute of Pharmaceutical Sciences.

SS Md Shafi's picture

Dear mekoo.. nice presentation. how it is helpful in cancer treatment ??

shafi ..

Guy-Armel BOUNDA's picture

Thank you Didier for your information concerning the Gene Therapy. This is very nice for us to understand the types if Therapy we can have access to. I am really looking forward to your next next blog as you will provide us information relating to Gene Therapy and Cancer. Keep it high!!!!!!!!!!!! Blessings

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