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Article: VIRUSES DELIVER DNA EFFICIENTLY, BUT . . . NONVIRAL GENE THERAPY VECTORS COMING UP FAST, BUT HELD BACK BY CELL TOXICITY; MIT CLAIMS FIX.
- Article from:
- BIOWORLD Today
- Article date:
- January 24, 2001
- Author:
CopyrightCOPYRIGHT 2001 A Thomson Healthcare Company. This material is published under license from the publisher through the Gale Group, Farmington Hills, Michigan. All inquiries regarding rights should be directed to the Gale Group. (Hide copyright information)
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Gene therapy - the clinical touchstone of biotechnology - scored its first success 10 years ago. In September 1990, a 4-year-old girl born with adenosine deaminase (ADA) deficiency, which crippled her immune system, received ADA replacement genes, delivered by a retroviral vector. That trailblazing experiment partially corrected her condition, as it did for a second small ADA victim, treated in February 1991.
Today, some 400 gene therapy clinical trials are under way worldwide.
Along with increasing technical sophistication, their practitioners confront a spectrum of roadblocks. Notably, the expanding toolkit of gene-delivery vectors, still primarily ...