Journal article Open Access

T Lymphocyte-Directed Gene Therapy for ADA-SCID: Initial Trial Results After 4 Years

Blaese, R. Michael; Culver, Kenneth W.; Miller, A. Dusty; Shearer, G.; Carter, Charles S.; Clerici, M.; Tolstoshev, P.; Fleisher, Thomas; Chang, L.; Rosenberg, S. A.; Chiang, Y.; Mullen, C. A.; Greenblatt, J. J.; Ramsey, W. J.; Klein, H.; Muul, L.; Berger, M.; Morgan, R. A.; Anderson, W. F.

In 1990, a clinical trial was started using retroviral-mediated transfer of the adenosine deaminase (ADA) gene into the T cells of two children with severe combined immunodeficiency (ADA− SCID). The number of blood T cells normalized as did many cellular and humoral immune responses. Gene treatment ended after 2 years, but integrated vector and ADA gene expression in T cells persisted. Although many components remain to be perfected, it is concluded here that gene therapy can be a safe and effective addition to treatment for some patients with this severe immunodeficiency disease.

Files (2.0 MB)
Name Size
2.0 MB Download
Views 473
Downloads 239
Data volume 469.2 MB
Unique views 421
Unique downloads 228


Cite as