title: Gene replacement rescues severe muscle pathology and prolongs survival in myotubularin-deficient mice and dogs.
authors.affiliation: 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.
authors.forename: Martin K
authors.initials: MK
authors.lastname: Childers
authors.affiliation: 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.
authors.forename: Alan H
authors.initials: AH
authors.lastname: Beggs
authors.affiliation: 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.
authors.forename: Ana
authors.initials: A
authors.lastname: Buj-Bello