Gene Therapy

Gene Therapy

This article deals with ‘Gene Therapy – UPSC.’ This is part of our series on ‘Science and Technology’ which is important pillar of GS-3 syllabus . For more articles , you can click here

What is Gene Therapy?

Using gene manipulating techniques to treat genetic diseases by replacing defective gene with healthy gene  or bolstering the immunity of body.

Types of Gene Therapy

It can be of two types

Somatic Cell Gene Therapy – Gene Therapy in normal cells
– Person will be treated for particular disease
Germ Cell Gene Therapy – Gene Therapy in Germline Cells
– For next generation ie Next generation will not be affected by genetic disorder


Same techniques which we used in Gene Editing like CRISPR Cas 9 etc (it is one of the application of Gene Editing only)

Gene Therapy

Some real examples

Yescarta – To treat blood cancer (Acute Lymphoblastic Leukemia)
– Yescarta is Gene Therapy to treat it.
– Developed by Novartis  

Side Note : It is given designation of Orphan Drug . Orphan Drug is  medicine that is intended to treat diseases so rare that sponsors are reluctant to develop them under usual marketing conditions.


  • Same as Gene Editing
    • Efficacy Issues
    • Side effects of virus delivery tools
    • Inclusivity issues since they are expensive
    • Playing with god (science vs conservatives)
    • Issues in case of Multi gene disorders and effect of the environment
  • Short lived nature of gene therapy : Patients will have to undergo multiple rounds of gene therapy. 
  • Immune response : Anytime a foreign object is introduced into human tissues, the immune system has evolved to attack the invader.
  • Grey areas in treatment : The treatment of human diseases through gene therapy for solely medical purpose is argued to be correct, however enhancement of human reproductive cells or altering/improving a normal person by gene manipulation are controversial areas as it may turn mankind into commodity.
  • Equal Access to treatment – The gene therapy at present has high cost

Leave a Comment