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Article: Findings on gene therapy and inherited disease presented at society meeting.
- Article from:
- Drug Week
- Article date:
- July 4, 2003
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2003 JUL 4 - (NewsRx.com & NewsRx.net) -- One of the most common forms of severe combined immune deficiency (SCID), also known as "bubble boy" disease, is adenosine deaminase (ADA) deficiency. A study presented at the annual meeting of the American Society of Gene Therapy demonstrated a new approach to treating patients with ADA-SCID.
ADA-SCID is treated through a bone marrow transplant from the parent with the best immunologic match or by injections of purified ADA enzyme. However, a good match may not be available, the transplant not successful, or enzyme replacement therapy may not generate enough T and B cells. ADA-SCID has been shown to be a prime target for ...