Study conducted in hamsters with same genetic defect as human muscular dystrophy shows great promise for treating adult heart failure
University of Pittsburgh investigators have for the first time used gene therapy to successfully treat heart failure and other degenerative muscle problems in an animal model that is genetically susceptible to a human muscular dystrophy. Reporting in the Oct. 25 edition of the journal Circulation, the authors say that this is the first successful attempt to deliver a therapeutic gene throughout the body.
"Previous attempts at systemic gene therapy for muscle have not been very effective because blood vessel capillaries act much like a mosquito net, blocking the gene drugs from reaching the muscle cells. Fortunately, we found a virus that is just small and sneaky enough to get through this net and deliver the therapeutic gene to both skeletal and cardiac muscle cells," said lead author, Xiao Xiao, Ph.D., associate professor of orthopaedic surgery at the University of Pittsburgh School of Medicine.
Jim Swyers | EurekAlert!
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