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

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 ther...

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Publicat a:Mol Ther
Autors principals: 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, Childers, Martin K.
Format: Artigo
Idioma:Inglês
Publicat: American Society of Gene & Cell Therapy 2017
Matèries:
Original Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC5383631/
https://ncbi.nlm.nih.gov/pubmed/28237839
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2017.02.004
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