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Gene replacement rescues severe muscle pathology and prolongs survival in myotubularin-deficient mice and dogs. | LitMetric
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Gene replacement rescues severe muscle pathology and prolongs survival in myotubularin-deficient mice and dogs.

Martin K Childers Alan H Beggs Ana Buj-Bello

Ann Transl Med

1 Department of Rehabilitation Medicine, 2 Institute for Stem Cell and Regenerative Medicine, School of Medicine, University of Washington, Seattle, Washington 98109, USA ; 3 The Manton Center for Orphan Disease Research, Division of Genetics and Genomics, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA ; 4 Généthon, INSERM UMR 951, Evry, France.

Published: October 2015

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 Source
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4620094PMC
http://dx.doi.org/10.3978/j.issn.2305-5839.2015.10.01DOI Listing

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