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?
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.
Cystic fibrosis
Haemophilia
Thalassemia
Severe Combined Immunodeficiency Disease
Genetic Disorders