WEDNESDAY, Jan. 28 (HealthDay News) -- Gene therapy with non-myeloablative conditioning shows promise in treating patients with a fatal disorder: severe combined immunodeficiency due to the lack of adenosine deaminase, according to a report published in the Jan. 29 issue of the New England Journal of Medicine.

Alessandro Aiuti, M.D., of the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy, and colleagues studied 10 children who were infused with autologous CD34+ bone marrow cells transduced with a retroviral vector containing the adenosine deaminase gene after undergoing non-myeloablative conditioning with busulfan.

After a median follow-up of four years, the researchers found that all 10 patients were still alive and that transduced hematopoietic stem cells engrafted and differentiated into myeloid cells containing adenosine deaminase and lymphoid cells. They also found that nine patients had immune reconstitution that allowed them to lead a normal life. Serious adverse events included prolonged neutropenia, hypertension, central-venous-catheter-related infections, Epstein-Barr virus reactivation and autoimmune hepatitis, the authors note.

"The prospects for continuing advancement of gene therapy to wider applications remain strong," state the authors of an accompanying editorial. "Ongoing and upcoming clinical trials will use safer designs of retroviral vectors, newer types of vectors for viral gene delivery, and emerging methods for direct in situ gene repair. These approaches to the treatment of hemoglobinopathies, hemophilia, muscular dystrophy, congenital retinopathies, neurodegenerative disorders, and other genetic diseases may further fulfill the promise that gene therapy made two decades ago."

One author reports being the chief of the board and chief executive officer of MolMed.

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