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Antibiotics Show Promise as Therapy for Genetic Disorders
Joan Stephenson, PhD
JAMA. 2001;285:2067-2068.
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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).
Furthermore, a growing body of research by the UAB group and others indicates that drugs such as gentamicin may one day be used to treat a subset of patients with a variety of genetic disorders, such as cystic fibrosis and muscular dystrophy. Although clinical trials testing gentamicin or agents with similar properties are at least a few years off, researchers say that laboratory and animal studies, as well as some pilot studies in human subjects, suggest that the . . . [Full Text of this Article]
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