A study appearing in Science Translational Medicine puts scientists one step closer to clinical trials to test a gene delivery strategy to improve muscle mass and function in patients with certain degenerative muscle disorders. Researchers at Nationwide Children’s Hospital have shown that a gene delivery strategy that produces follistatin - a naturally occurring protein that inhibits myostatin, a growth factor expressed specifically in skeletal muscle - directly to the quadriceps of non-human primates results in long-term gene expression with muscle enhancing effects, including larger muscles with greater strength. Jerry Mendell, MD, principal investigator in the Center for Gene Therapy at Nationwide Children’s added, "These findings serve as the basis for testing in patients and give us confidence in moving forward with our translational program of follistatin to enhance muscle mass. The potential use of this strategy for muscle strengthening has important implications for patients with muscle diseases including Duchenne muscular dystrophy - the most common form of muscular dystrophy - as well as for the planned first clinical trial for inclusion body myositis. Country [Not specified] Albania Algeria Argentina Armenia Australia Austria Azerbaijan Bahrain Bangladesh Belarus Belgium Belize Bolivia Bosnia and Herzegovina Brazil Brunei Darussalam Bulgaria Canada Caribbean Chile Colombia Costa Rica Croatia Czech Republic Denmark Dominican Republic Ecuador Egypt El Salvador Estonia Faroe Islands Finland France Georgia Germany Greece Guatemala Honduras Hong Kong S. Macedonia (FYROM) Malaysia Maldives Malta Mexico Mongolia Morocco Netherlands New Zealand Nicaragua Norway Oman Panama Paraguay People’s Republic of China Peru Philippines Poland Portugal Principality of Monaco Puerto Rico Qatar Republic of the Philippines Romania Russia Saudi Arabia Serbia Singapore Slovakia Slovenia South Africa Spain Sweden Switzerland Syria Taiwan Thailand Trinidad and Tobago Tunisia Turkey U. read more
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