Monday, January 27, 2020

Gene Therapy Shows Promise for Rare Immune Disease

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Monday, January 27, 2020

Gene Therapy Shows Promise for Rare Immune Disease

Findings from early-stage clinical trials suggest the promise of a gene therapy approach for repairing immune function in people with X-linked chronic granulomatous disease (CGD), a genetic immune deficiency characterized by susceptibility to certain bacterial and fungal infections. An international team including NIAID scientists reports results from nine gene therapy recipients, aged 2 to 27 years, today in Nature Medicine. One year after receiving the gene therapy, seven of the participants had no new CGD-related infections, and six were able to stop taking the daily prophylactic antibiotics needed to reduce risk from the life-threatening infections that can occur in people with CGD.

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