The latest advances in such areas as gene therapy and stem cell biology are putting medical science closer to finding options for people with as-yet incurable muscle conditions that cause disability and shorten lives. A few of these disorders eventually result in neuromuscular breathing weakness or failure requiring mechanical ventilation.
“What the new muscle research center offers patients with these diseases, many of whom are racing against time, is hope, “said center director Michael Regnier, professor of bioengineering, a jointly operated department of the University of Washington College of Engineering and the UW School of Medicine.
A few of the several diseases for which the new center initially will be seeking answers are
- amyotrophic lateral sclerosis, or ALS, whose cause is unknown and which leads to progressive muscle weakness and eventually to breathing and swallowing problems,
- The muscular dystrophies, a group of genetically inherited, progressively debilitating disorders in which a muscle protein is missing or not made correctly. This work will be done in partnership with related disease-research programs, such as the UW Medicine Wellstone Muscular Dystrophy Center.
- Myotubular myopathy, a congenital disease of varying severity that causes problems with the tone and contraction of muscles.
Time is of the essence for many patients eagerly awaiting treatment progress in muscle diseases that are characterized by a decline and weakening.
Clinical partners include pediatric experts in rare genetic disorders and physical medicine physicians who care for patients with neuromuscular disorders at the UW Medical Center Rehabilitation Medicine Clinic and other settings.
One of several areas in which the center is expected to take a major leadership role is in growing human stem cells, some of which will be derived from patient cells, to produce disease-in-a-lab-dish models. These models will improve understanding of how the disease pathology begins and develops, and will also serve for testing possible treatments.
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