Gene Therapy

Gene therapy for muscular dystrophy shows promise beyond safety

Published in Genetics and Environmental Business Week, April 30th, 2009

Researchers have cleared a safety hurdle in efforts to develop a gene therapy for a form of muscular dystrophy that disables patients by gradually weakening muscles near the hips and shoulders.

Described as the first gene therapy trial in muscular dystrophy to demonstrate promising findings, researchers from the University of Florida, Nationwide Children's Hospital in Columbus, Ohio, and The Ohio State University report how they safely transferred a gene to produce a protein necessary for healthy muscle fiber growth into three teenagers with limb-girdle muscular dystrophy.

The findings, which have relevance to genetic disorders beyond muscular...

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Source: Genetics and Environmental Business Week (2009-04-30)

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