Gene Research
Genetic disorder that causes severe immunodeficiency may be cured by in vivo gene therapy technique
Published in Gene Therapy Weekly, June 8th, 2006
In-vivo gene therapy successfully restored the immune system in basset pups with X-linked severe combined immunodeficiency, or XSCID, a life-threatening genetic disorder that effectively disables the immune system. Researchers from the University of Pennsylvania School of Veterinary Medicine and the National Institute of Allergy and Infectious Disease injected a retrovirus containing a corrective version of the gene responsible for XSCID, an important proof of principle for the technique of "in-vivo" gene therapy.
Their findings are presented in a recent issue of the journal, Blood.
In humans, XSCID affects one in 100,000 boys, resulting from...
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Source: Gene Therapy Weekly (2006-06-08)
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