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

Favorable interim results reported in stem-cell therapy trial

January 8, 2018 | Categories: Research | Tagged: , , ,

A therapy developed at UW shows promise in its first test in young boys who have an inherited deadly muscle disease. Favorable interim results have been announced in a small clinical trial of a stem-cell therapy to treat patients with a rare, deadly disease called myotubular myopathy. 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

New book published on regenerative medicine for degenerative muscle diseases

January 12, 2016 | Categories: Announcements | Tagged: , , , , ,

Congratulations to ISCRM’s Casey Childers and Hannele Ruohola-Baker who are contributing authors of a new book entitled Regenerative Medicine for Muscle Diseases. The book, edited by Casey Childers, delves into the cutting-edge research having to do with degenerative skeletal disorders, such as Duchenne muscular dystrophy and congenital myopathy, and gene replacement therapy and stem cell […] Read More

ISCRM Faculty Member featured in The Scientist

December 1, 2015 | Categories: Announcements | Tagged: , , ,

The work of ISCRM faculty member, Casey Childers, on a gene therapy in children with a rare and fatal muscle weakness disorder called X-linked myotubular myopathy was highlighted in a Dec. 1, 2015 article in The Scientist. The piece looks at cell therapies approaching the clinic. Read the full story at The Scientist. Read More