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