Genetic Therapy

Advent of Recombinant DNA Technology has made it possible to detect defective genes and replace them with “good” genes. Consequently the genetic diseases, which result on mutations can now be cured. The first successful gene therapy experient was done on a 9 year old Indian girl Ashanti in 1990 in USA, who was suffering from a fatal immunological disease – SCID, which is caused by a mutation in ADA gene. The scientists introduced the the “good” copy of the gene in her body and were successful in curing her of this fatal disease. This encouraged other researchers and now several methods are being tried to cure genetic diseases. The 21st Century is the era of Gene therapy and keeping in view tremendous advancements made in the Recombinant DNA technology, it is expected that cure for several genetic defects would be available within the next decade.

Some Expert Comments:

• The detection of defective genes and their replacement works quite well in vitro, but not in-vivo.
• There are no cures to genetic disorders based on mutations. I assure you this is incorrect; there has not been a single case of gene therapy curing a genetic disorder. Efforts to transduce normal copies of genes into patients with genetic disorders have been successful to varying degrees, but no cures yet.
• In Ashanta’s story, there was no cure – simply incorrect. It was nonetheless a hallmark in biomedical research; it hinted at the promise gene therapy held.
• As far as the claim that genetic therapy may cure several disorders in the next decade, only time will tell. I would not venture to be so prophetic. A few years ago Harold Varmus, then director of the National Institutes of Health, assembled a panel of experts to assess the status of the field of gene therapy. You see, for quite some time many in the field had been feeling that gene therapy was being pushed to the patient’s bed prematurely. The panel’s conclusion was right on the mark: the field needs much more research at the fundamental levels, such as development of safe, efficient, and specific gene delivery vectors.