1.

a. Name the deficiency for which first clinical gene therapy was given.b. Mention the causes of and one cure for this deficiency.

Answer»

a. Adenosine deaminase deficiency (ADA).

b.

Cause: Deletion of ADA gene.

Cure: Bone marrow transplantation/enzyme replacement therapy/giving functional ADA to patient by injection/infusion of genetically engineered lymphocytes/introducing gene isolated from marrow cells producing ADA into cells at early embryonic stages.



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