Question

Suggest a possible permanent remedy for ADA deficiency.

Answer 1

• Gene therapy is a possible permanent remedy for ADA (Adenosine Deaminase) deficiency. Replacing the defective ADA gene with a functional one, enabling the patient to produce the enzyme naturally.
• This treatment can potentially restore normal immune function permanently and reduce or eliminate the need for ongoing treatments, such as enzyme replacement or lymphocyte infusions.
• If the gene taken from bone marrow cells that make ADA is put into cells early in the embryonic stage, a lasting cure might be possible.