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
Favorable interim results reported in stem-cell therapy trial
January 8, 2018 | Categories: Research |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
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
New book published on regenerative medicine for degenerative muscle diseases
January 12, 2016 | Categories: Announcements |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 |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