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

Gene replacement therapy that works in dogs to be tested in children

May 18, 2017 | Categories: Research | Tagged: , , , ,

SEATTLE – KIRO 7 has learned of a revolutionary treatment, here in Seattle, that has saved the lives of dogs and is just months away from being tested in children. In video obtained by KIRO 7, dogs that were destined to die show no signs of the disease after a single infusion of gene-replacement therapy.  The […] 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

UW, family rewriting neuromuscular research

November 23, 2016 | Categories: Announcements | Tagged: , ,

Physicians and researchers at the UW Medicine Institute for Stem Cell and Regenerative Medicine have partnered up with a nonprofit foundation in one of the leading efforts to find a cure or treatment for a rare neuromuscular disease called myotubular myopathy (MTM). The inherited disease is almost exclusively found in males and becomes evident at birth. […] 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

Childers displays a dog family tree showing those affected and unaffected by an inherited muscle disorder similar to X-linked myotubular myopathy in people.

Gene therapy leads to robust improvements in animal model of fatal muscle disease

January 22, 2014 | Categories: Research | Tagged: , ,

January 22, 2014 Preclinical studies show that gene therapy can improve muscle strength in small- and large-animal models of a fatal congenital childhood disease know as X-linked myotubular myopathy. The findings, appearing  as the cover story in the January 22, 2014 issue of Science Translational Medicine, also demonstrate the feasibility of future clinical trials of gene therapy for […] Read More