Question

The first clinical gene therapy was given for treating

• A. Diabetes mellitus
• B. Chickenpox
• C. Rheumatoid arthritis
• D. Adenosine deaminase deficiency

Answer 1

Answer: (D)

Adenosine deaminase deficiency

• The first human gene therapy trial was conducted at the National Institutes of Health in Bethesda, Maryland, in 1990 on a four-year-old girl suffering from ADA deficiency.
• A functional ADA gene was introduced ex vivo in bone marrow cells of the child through an engineered retrovirus containing a functional ADA gene. The treated cells were reintroduced into the patient’s marrow.

So, the correct answer is option D.