Two men wearing white lab coats stand and smile in a laboratory. Shelves with lab equipment and supplies are visible in the background, and a window lets in natural light.

New Gene Therapy for Muscular Dystrophy Offers Hope

July 17, 2024 | Categories: Core Faculty, Research | Tagged: , ,

A new gene therapy treatment for Duchenne muscular dystrophy (DMD) shows promise of not only arresting the decline of the muscles of those affected by this inherited genetic disease, but perhaps, in the future, repairing those muscles. Read More

Diagram showing an XLMTM dog transcriptome leading to rAAV9-cMTM1 gene transfer, resulting in two outcomes: a healthy dog labeled RESCUED with therapeutic effect and a sick dog labeled RESISTANT with sub-therapeutic effect.

New Insights from Mack Lab on Mechanisms of Gene Therapy for X-Linked Myotubular Myopathy

January 27, 2020 | Categories: Research | Tagged: , ,

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Three microscopic images of mouse heart muscle tissue, labeled a, b, and c, with text describing the results of micro-dystrophin gene therapy for Duchenne muscular dystrophy. Images show green-stained muscle fibers with varying patterns.

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 […] Read More

A man in a white lab coat kneels next to a black dog on a leash in a white-walled room with colorful padded blocks and a yellow mat on the floor.

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 […] Read More

Book cover with a blue background. Title: Regenerative Medicine for Degenerative Muscle Diseases. Editor: Martin K. Childers. Series title at the top: Stem Cell Biology and Regenerative Medicine.

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 […] Read More

People sitting at outdoor tables and benches between two modern glass buildings on a sunny day, with string lights hanging above and greenery along the walkway.

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, […] Read More