The disease not treated by Ex-vivo gene therapy is:

Correct answer: C. Hypercholesterolemia

  • A. SCID
  • B. Diabetes
  • C. Hypercholesterolemia
  • D. All of the above

Explanation

Among the many gene therapy trials, one is for the treatment of familial hypercholesterolemia a condition that develops when liver cells lack a receptor for removing cholesterol from the blood. The high levels of blood cholesterol make the patient subject to fatal heart attacks at a young age. In a newly developed procedure, a small portion of the liver is surgically excised and infected with a retrovirus containing a normal gene for the receptor. Several patients have experienced a lowering of serum cholesterol levels following this procedure

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