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Medical News & Perspectives
April 25, 2001

Antibiotics Show Promise as Therapy for Genetic Disorders

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JAMA. 2001;285(16):2067-2068. doi:10.1001/jama.285.16.2067-JMN0425-2-1

New research findings involving a tried-and-true antibiotic developed decades ago are pointing the way toward a novel approach for treating genetic disorders in the 21st century, researchers suggest.

In one recent study, investigators at the University of Alabama at Birmingham (UAB) found evidence that gentamicin treatment enabled cells from patients with Hurler syndrome to override the effects of the mutation underlying the disease (Hum Mol Genet. 2001:10:291-299).