Question

A patient with ADA deficiency requires periodic infusion of genetically engineered lymphocytes. Explain why such periodic infusion is required and also suggest a permanent cure for such ADA deficiency.

Answer 1

Because these cells have a fixed lifespan because lymphocytes do not self-replicate endlessly, a patient with ADA (adenosine deaminase) deficiency needs to receive periodic infusions of genetically modified lymphocytes. The long-term solutions are gene therapy at the embryonic stage (before birth) or bone marrow transplantation (if a suitable donor is available).