Gene Therapy Cassettes Improved for Muscular Dystrophy
February 1, 2019 | Categories: Research |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 gene is called, has been restructured to enhance its functionality. The revamped versions were developed and tested at […] Read More
Gene therapy treats muscle-wasting disease in dogs
February 15, 2017 | Categories: Research |Work on gene therapy is showing significant progress for restoring muscle strength and prolonging lives in dogs with a previously incurable, inherited neuromuscular disease. UW Medicine Institute for Stem Cell and Regenerative Medicine scientists are leading the multi-institutional research effort. The disease arises from a mutation in genes that normally make a protein, called myotubularin, […] Read More
Mutations in genes that modify DNA packaging result in form of muscular dystrophy
November 19, 2012 | Categories: Research |November 19, 2012 A recent finding by medical geneticists sheds new light on how facioscapulohumeral muscular dystrophy develops and how it might be treated. More commonly known as FSHD, the devastating disease affects both men and women. FSHD is usually an inherited genetic disorder, yet sometimes appears spontaneously via new mutations in individuals with no […] Read More