## **Core Concept**
The question revolves around the historical milestone of gene therapy, specifically focusing on the first successful application of somatic enzyme gene therapy. Gene therapy involves making targeted changes to a person's genome to treat or cure diseases. Somatic enzyme gene therapy targets non-reproductive cells to correct a genetic deficiency.
## **Why the Correct Answer is Right**
The first successful gene therapy was performed to treat **Severe Combined Immunodeficiency (SCID)**, specifically the adenosine deaminase (ADA) deficiency form. This condition leads to a severe impairment of the immune system. The therapy aimed to introduce a functional ADA gene into the patient's somatic cells, thereby enabling the production of the ADA enzyme and improving immune function. This pioneering work was conducted by **William French Anderson** and his team in 1990.
## **Why Each Wrong Option is Incorrect**
- **Option A:** Not specified due to lack of information.
- **Option B:** Not specified due to lack of information.
- **Option D:** Not specified due to lack of information.
## **Clinical Pearl / High-Yield Fact**
A crucial point to remember is that the first gene therapy was for **ADA deficiency**, a form of SCID. This historic treatment marked the beginning of gene therapy as a potential treatment for genetic diseases. The patient, **Ashanti DeSilva**, received modified T cells with the ADA gene, showing the feasibility of gene therapy in correcting genetic defects.
## **Correct Answer:** C. Severe Combined Immunodeficiency (SCID) due to ADA deficiency.
Free Medical MCQs Β· NEET PG Β· USMLE Β· AIIMS
Access thousands of free MCQs, ebooks and daily exams.
By signing in you agree to our Privacy Policy.