Gene therapy defeats muscle disease in tests

Injections rescue mice, dogs from fatal affliction, establish feasibility for human trials

By Kim Blakeley  |  UW Today  |  Updated 11:00 AM, 01.22.2014

Posted in: Research

  • Two Labs one year post-treatment show no signs of the muscle disease. Martin Childers

Preclinical studies show that gene therapy can improve muscle strength in small- and large-animal models of a fatal congenital childhood disease know as X-linked myotubular myopathy. 

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Tagged with: gene therapy, animal testing
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