Gene Therapy for Adenosine Deaminase Deficiency

Gene therapy with nonmyeloablative conditioning is safe and effective for patients with severe combined immunodeficiency caused by lack of adenosine deaminase.

Adenosine deaminase (ADA) deficiency is an ultimately fatal disorder characterized by multiple complications, many of which are infectious in origin. Early attempts at gene therapy for ADA deficiency have had some success but have not resulted in immune-system reconstitution. Now, in a study with partial industry support, a multinational team of investigators has tried a novel approach.

The trial involved 10 children with ADA deficiency who lacked an HLA-identical sibling donor. After nonmyeloablative conditioning with busulfan, the children received infusions of autologous CD34⁺ bone marrow cells that had been transduced with a retroviral vector containing the ADA gene. No enzyme-replacement therapy was given following infusion of these cel…

Reviewing Author

Larry M. Baddour, MD

Disclosures

RoyaltiesUpToDate, Inc. (Authorship duties)

Larry M. Baddour, MD

Disclosures

RoyaltiesUpToDate, Inc. (Authorship duties)