New gene transfer strategy shows promise for limb girdle and other muscular dystrophies
Monday, July 9, 2012 - 10:00
in Biology & Nature
The challenge of treating patients with genetic disorders in which a single mutated gene is simply too large to be replaced using traditional gene therapy techniques may soon be a thing of the past. A new study describes a new gene therapy approach capable of delivering full-length versions of large genes and improving skeletal muscle function. The strategy may hold new hope for treating dysferlinopathies and other muscular dystrophies.