What is gene therapy ? Illustrate using the example of adenosine deaminase (ADA) deficiency.
What is gene therapy ? Illustrate using the example of adenosine deaminase (ADA) deficiency.

The technique of introducing DNA into an organism, such as humans, in order to treat a disease is known as gene therapy. It’s utilised to replace a gene product that’s been lost or to rectify mutant alleles. Adenosine deaminase deficiency (ADA) is an autosomal-recessive hereditary condition caused by a faulty adenosine deaminase (ADA) enzyme. 

Please note that people with this enzyme deficiency have severe combined immunodeficiency (SCID). A human gene therapy study could be utilised to ex vivo introduce a functioning ADA gene into SCID patients’ bone marrow cells. The ADA gene is transferred into stem cells derived from the patient with ALS using a modified retrovirus containing a functioning ADA gene.