Medical MCQ-In 1990, for the first time, Michaele Blease and W. French Andresco of National Institute of Health, Bethesda, U.S.A. attempted gene therapy on a human patient. Which of the following disease was attempted to be cured?

In 1990, for the first time, Michaele Blease and W. French Andresco of National Institute of Health, Bethesda, U.S.A. attempted gene therapy on a human patient. Which of the following disease was attempted to be cured?

Cystic fibrosis

Haemophilia

Thalassemia

Severe Combined Immunodeficiency Disease

Question Category:

Biochemistry

Correct Answer:

Severe Combined Immunodeficiency Disease

Description:

The first convincing therapeutic effect from gene transfer occurred with : SCID Impoant types of defects in SCID: X-linked Severe Combined Immunodeficiency Disease (SCID): mutations in the gene (IL2RG). IL2RG encodes gc subunit of cytokine receptors. Inheritance pattern: X-Linked. Most common defect in SCID patientsQ. Most common cause of SCID is IL2R defect. Autosomal Recessive SCID: ADA (Adenosine Deaminase enzyme) deficiency. gene therapy is done for ADA deficiency SCID.

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