One successful form of gene therapy has involved delivery of an allele for the enzyme adenosine deaminase (ADA)to bone marrow cells of a child with SCID, and delivery of these engineered cells back to the bone marrow of the affected child. What is one major reason for the success of this procedure as opposed to many other efforts at gene therapy?

Question

Quizplus · Accepted Answer

The engineered cells are designed to home back to the bone marrow when reintroduced into the patient, providing a direct and effective way to replace the dysfunctional cells with functional ones. This targeted delivery is a key factor in the success of this gene therapy approach for SCID.

One Successful Form of Gene Therapy Has Involved Delivery of an Allele