Q11 of 30 Page 1

Name the disease that was first to get the gene therapy treatment. Write the cause of the disease and the effect it has on the patient.

Gene therapy treatment was 1st used to treat SCID (Severe combined immune deficiency) disease.


The SCID patient has a defective gene for the enzyme ADA (Adenosine Deaminase) , due to which the patient lacks functional T-lymphocytes and therefore fails to fight the infecting pathogen.


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