Intravenous Administration of a MTMR2-Encoding AAV Vector Ameliorates the Phenotype of Myotubular Myopathy in Mice
in Journal of Neuropathology & Experimental Neurology
April 2018; p ublished online February 2018 .
Journal Article. Subjects: Neurology; Clinical Neuroscience; Pathology; Histopathology. 7886 words.
X-linked myotubular myopathy (XLMTM) is a severe congenital disorder in male infants that leads to generalized skeletal muscle weakness and is...