Written by – Suraj Kahar
Gene therapy is a therapeutic approach towards treatment of genetic disorders and diseases. The start of gene therapy can be dated back to 1972. Basically it’s the delivery of a functional gene inside the body, replacing the inactive or the damaged gene. Well gene therapy can be called as a boon for human society, but yes, it does have many flaws and ethical issues. Nevertheless gene therapy has been proved to cure many severe diseases like Severe Combined Immune Deficiency (ADA-SCID, Chronic Granulomatus Disorder (CGD), Hemophilia, Parkinson’s Disease, Huntington’s Disease etc.
Gene therapy has two major types:
- Somatic gene therapy: transfer of genes into somatic cells (organs).
- Germ line gene therapy: gene transfer into germ cells (sperm and ova).
Though this seems to be fascinating, germ line therapies can have a disastrous effect and can lead to mutations that will be permanent in that individual and will pass on to the next generations. Somatic therapies are almost with no dangers but their success rates are very low. Still people look forward towards these therapies because it completely treats the disorders, and generally one would prefer gene therapy instead of using costly drugs which have many side-effects and leads to microbial resistance.
BASIC OUTLINE OF GENE THERAPY
In in-vivo therapy, genes are modified in the cell, inside the body. The method involves use of ballistic gene gun, plasmid DNA, recombinant virus, DNA liposomes, tissue injection and systematic infusion.In ex-vivo therapy, cells of the patient are modified outside the body and transplanted back in the patient.Viral vectors are important since they are precise in gene delivery.
PROBLEMS ASSOCIATED WITH GENE THERAPY:
• Multiple rounds of treatment requied since it is short-lived
• Leads to immune response because a foreign object in introduced in the body.
• Can cause a disease since viral vectors are used.
• Some cases have seen multi-gene disorders due to insertion.
All these above mentioned problems can be easily solved and this could make the therapy much advanced and better.
As many of these methods are still in Phase 3 & 4 clinical trials, modification and enhancement will make it safe. Gene therapy is the future of medicine industry and will be a boon for humans.