Gene therapy reverses effects of lethal childhood muscle disorder in mice

Reversing a protein deficiency through gene therapy can correct motor function, restore nerve signals and improve survival in mice that serve as a model for the lethal childhood disorder spinal muscular atrophy.

Eurekalert: http://www.eurekalert.org/pub_releases/2010-02/osu-sg022610.php

Reference

Published in Nature Biotechnology Published online 28 February 2010