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    • Systemic AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in Dogs 

      Mack, David L.; Poulard, Karine; Goddard, Melissa A.; Latoumerie, Virginie; Snyder, Jessica M.; Grange, Robert W.; Elverman, Matthew R.; Denard, Jerome; Veron, Philippe; Buscara, Laurine; Le Bec, Christine; Hogrel, Jean-Yves; Brezovec, Annie G.; Meng, Hui; Yang, Lin; Liu, Fujun; O'Callaghan, Michael; Gopal, Nikhil; Kelly, Valerie E.; Smith, Barbara K.; Strande, Jennifer L.; Mavilio, Fulvio; Beggs, Alan H.; Mingozzi, Federico; Lawlor, Michael W.; Buj-Bello, Ana; Childers, Martin K. (2017-04-05)
      X-linked myotubular myopathy (XLMTM) results from MTM1 gene mutations and myotubularin deficiency. Most XLMTM patients develop severe muscle weakness leading to respiratory failure and death, typically within 2 years of ...