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- Title
Points of Light in Gene Therapy Renaissance.
- Authors
LEWIS, RICKI
- Abstract
The article provides success stories about patients subjected to gene therapy. The case of 9-year old Corey Haas who was headed for blindness from Leber congenital amaurosis type 2 (LCA2) and the nature of the disease and how it was addressed by gene therapy are discussed. Problems relating to gene therapies for severe combined immune deficiency (SCID) and new trials using the safer "self-inactivating" retroviral vectors are discussed. Treatment of adrenoleukodystrophy (ALD) is explained in contrast with stem cell transplant.
- Subjects
PATIENTS; GENE therapy; HAAS, Corey; BLINDNESS; SEVERE combined immunodeficiency
- Publication
Bio-IT World, 2010, Vol 9, Issue 4, p13
- ISSN
1538-5728
- Publication type
Article