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Question

First gene therapy was performed for the disease:

A
Down’s syndrome
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B
ADA deficiency
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C
Cystic fibrosis
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D
Alkaptonuria
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Solution

The correct option is B ADA deficiency
The first clinical gene therapy was performed in 1990 on a 4-year old girl for treatment of a genetic defect that left her with ADA-SCID, a severe immune system deficiency.

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