Gene Therapy

Last Updated: May 2023 (Gene Therapy)

Gene Therapy

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


Gene Therapy
  • Gene Therapy means using gene manipulating techniques to treat diseases by replacing the defective gene with a healthy gene or boosting the body’s immunity.
  • It is an extremely useful tool for numerous diseases, like severe combined immune deficiencies, haemophilia, Parkinson’s disease, cancer, etc.

Types of Gene Therapy

Gene Therapy can be of two types

Somatic Cell Gene Therapy

It involves Gene Therapy in normal cells. 

It has two categories:

  1. Ex-Vivo: Cells are taken from an individual, modified genetically outside the body and then transplanted into the same person’s body or some other person.
  2. In-Vivo: Genetic modification is done inside the body using a gene delivery system.

Germ Cell Gene Therapy

  • Germ Cell Gene Therapy is carried out in Germline Cells (sperm or egg).
  • Next-generation will not be affected by a genetic disorder.


  • Same techniques which we used in Gene Editing, like CRISPR Cas9. (Gene Therapy is one of the applications of Gene Editing only).
Techniques of Gene Editing

Some real examples

#1. Yescarta

  • Yescarta is Gene Therapy developed by Novartis to treat blood cancer (Acute Lymphoblastic Leukaemia). 

#2. Zolgensma

  • Gene Therapy developed by Novartis to treat Muscular Atrophy.
  • It is extremely expensive, costing over 15 crore.

Benefits of Developing Gene Therapy for India

  • In India, around seven crore people suffer from genetic diseases such as Thalassemia, Sickle Cell Anaemia etc. These can be cured using Gene Therapy.
  • Economic benefits: It is a growing market with a potential market of $250 billion. India can earn huge revenue and strengthen its position as a destination for medical tourism by developing expertise.

Problems with Gene Therapy

  • The efficacy of Gene Therapy is still questioned. 
  • Short-lived nature of gene therapy as a result of which patients have to undergo multiple rounds of gene therapy. 
  • Immune response: The immune system has evolved to attack the invader whenever a foreign object is introduced into human tissues. Hence, when foreign DNA is introduced into the body for gene therapy, the immune system attacks it and reduces the efficacy of the gene therapy.
  • Side effects of virus delivery tools: Viruses are used as vectors in most gene therapies. This virus can cause various problems for the patient undergoing gene therapy like toxicity, immunity and inflammatory responses, gene control, etc. 
  • Grey areas in treatment: The treatment of human diseases through gene therapy for solely medical purposes is argued to be correct. However, enhancing human reproductive cells or altering/improving an average person by gene manipulation are controversial areas as they may turn humanity into a commodity. 
  • Equal Access to treatment: Gene therapy at present has a high cost. Hence, there are inclusivity issues associated with gene therapy. 

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