Pediatrics Genetics And Genetic Disorders 4aca470c

Gene therapy is most commonly targeted against:

SCID

cancer

cystic fibrosis

Leukemia

High-Yield Explanation

a. SCID(Ref: Nelson's 20/e p 584-586)FDA-approved gene therapy is used for ADA-SCID, Leber's congenital amaurosis, multiple myeloma & Adrenoleukodystrophy

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