Systemic AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in Dogs
Systemic AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in Dogs
About this item
Full title
Author / Creator
Mack, David L. , Poulard, Karine , Goddard, Melissa A. , Latournerie, Virginie , Snyder, Jessica M. , Grange, Robert W. , Elverman, Matthew R. , Denard, Jérôme , 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 and Childers, Martin K.
Publisher
United States: Elsevier Inc
Journal title
Language
English
Formats
Publication information
Publisher
United States: Elsevier Inc
Subjects
More information
Scope and Contents
Contents
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 age. Our objective was to evaluate the efficacy and safety of systemic gene therapy in the p.N155K canine model of XLMTM by perfor...
Alternative Titles
Full title
Systemic AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in Dogs
Authors, Artists and Contributors
Author / Creator
Poulard, Karine
Goddard, Melissa A.
Latournerie, Virginie
Snyder, Jessica M.
Grange, Robert W.
Elverman, Matthew R.
Denard, Jérôme
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.
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_5383631
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_5383631
Other Identifiers
ISSN
1525-0016
E-ISSN
1525-0024
DOI
10.1016/j.ymthe.2017.02.004
