Cystic Fibrosis

Phase I Clinical Trial Indicates More Research Is Needed

Published in Gene Therapy Weekly, August 23rd, 2001

by Deborah W. Heinrich, PhD, staff medical writer - A Phase I clinical trial using gene therapy for the treatment of cystic fibrosis initiated by Genzyme Corporation revealed promising results but indicated that more research will be necessary for successful treatment.

M.A. Perricone and colleagues, Genzyme Corp., constructed a recombinant adenovirus vector expressing the cystic fibrosis trans-membrane regulator (Ad2/CFTR), which they administered to cystic fibrosis (CF) patients. Airway epithelial cells were evaluated for transfection by polymerase chain reaction (PCR) and CFTR expression by reverse transcriptase PCR (rtPCR). Transfection efficiency was evaluated by...

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Source: Gene Therapy Weekly (2001-08-23)

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