Gene therapy’s success in dogs spurs hope about human trial

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Gene therapy’s success in dogs spurs hope about human trial

May 22, 2017

Within months, clinical trials of a gene therapy will begin with a small group of children affected with a deadly muscle-wasting disease.

Dr. Martin Childers, a UW Medicine researcher and physician, has led development of a therapy to address a form of muscular dystrophy called myotubular myopathy. Boys affected with the disease often die before age 2.

Dogs who have received injections of the genetic therapy have achieved remarkable results. Once destined to die, dogs show no signs of the disease after a single infusion of gene-replacement therapy. 

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