Gene Therapy

Researchers from Columbus Children's Research Institute provide details of new studies and findings in the area of gene therapy

Published in Gene Therapy Weekly, October 18th, 2007

New research, 'Gene therapy for duchenne muscular dystrophy: expectations and challenges,' is the subject of a report. "Duchenne muscular dystrophy is a debilitating X-linked disease with limited treatment options. We examined the possibility of moving forward with gene therapy, an approach that demonstrates promise for treating Duchenne muscular dystrophy," investigators in the United States report.

"Gene therapy is not limited to replacement of defective genes but also includes strategies using surrogate genes with alternative but effective means of improving cellular function or repairing gene mutations. The first viral-mediated gene transfer for any muscle disease...

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Source: Gene Therapy Weekly (2007-10-18)

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