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Gene Therapy Cassettes Improved for Muscular Dystrophy


Experimental gene therapy cassettes for Duchenne muscular dystrophy have been modified to deliver better performance. The cassettes, which carry the therapy into muscle cells, contain newer versions of a miniaturized treatment gene. The micro-dystrophin, as the treatment is called, has been restructured to enhance its functionality.

The revamped versions were developed and tested at UW Medicine labs in animal models of muscular dystrophy.  The results will be published Feb. 1 in Molecular Therapy, a Cell Press journal. 

Duchene muscular dystrophy is a life-shortening genetic disorder characterized by debilitating muscle weakness that gets worse with time.  The condition almost exclusively affects males.  It’s caused by X chromosome mutations that interfere with the production of dystrophins, which build and maintain healthy muscles. 

Gene Therapy Cassettes Improved For Muscular Dystrophy