What is GENE THERAPY

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1. A new system of medicine gene therapy, may develop to treat some hereditary disease such as SCID, haemophilia etc.
2. It is the technique for curing genetic disease through replacing a “Faculty Gene” by a normal healthy functional gene.
3. The first gene therapy used in severe combined immune deficiency (SCID) Pateint.
4. Infact with SCID disorder lack the enzyme adenosine deaminase (ADA)
5. ADA enzyme involved in purine metabolism.
6. These patients have no functioning T & B lymphocytes.
7. The affected child of SCID develops recurrent infection early in life because they have no immune response against invading pathogen.
8. The ideal approach would be to give the patient a functioning ADA by gene therapy.
9. Thus, genetic disorder can be over come by introducing specific gene.

Author: Dr Anurag Jain
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