Gene Therapy Cassettes Improved for Muscular Dystrophy

February 1, 2019 | Categories: Research | Tagged: , , ,

February 1, 2019 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 […] Read More

Transfer of the MTM1 gene into the weakened muscle tissue of dogs affected by myotubular myopathy restores muscle strength. The disease arises from a genetic mutation that disrupts the production of a protein needed for muscle function.

Gene therapy treats muscle-wasting disease in dogs

February 15, 2017 | Categories: Research | Tagged: , , ,

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

Dr. Daniel Miller studies the molecular basis of muscle disorders in his lab at UW Medicine South Lake Union.

Mutations in genes that modify DNA packaging result in form of muscular dystrophy

November 19, 2012 | Categories: Research | Tagged: , , ,

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