作者：
David L,Mack [1]
;
Karine,Poulard [2]
;
Melissa A,Goddard [3]
;
Virginie,Latournerie [2]
;
Jessica M,Snyder [4]
;
Robert W,Grange [5]
;
Matthew R,Elverman [3]
;
Jérôme,Denard [6]
;
Philippe,Veron [2]
;
Laurine,Buscara [2]
;
Christine,Le Bec [6]
;
Jean-Yves,Hogrel [7]
;
Annie G,Brezovec [5]
;
Hui,Meng [8]
;
Lin,Yang [9]
;
Fujun,Liu [9]
;
Michael,O'Callaghan [10]
;
Nikhil,Gopal [11]
;
Valerie E,Kelly [12]
;
Barbara K,Smith [13]
;
Jennifer L,Strande [14]
;
Fulvio,Mavilio [2]
;
Alan H,Beggs [15]
;
Federico,Mingozzi [16]
;
Michael W,Lawlor [8]
;
Ana,Buj-Bello [17]
;
Martin K,Childers [18]
作者单位：
Department of Rehabilitation Medicine, University of Washington, Seattle, WA 98104, USA; Institute for Stem Cell and Regenerative Medicine, School of Medicine, University of Washington, Seattle, WA 98107, USA.
[1]
Genethon, 91000 Evry, France; INSERM, UMR_S951, 91002 Evry, France.
[2]
Institute for Stem Cell and Regenerative Medicine, School of Medicine, University of Washington, Seattle, WA 98107, USA.
[3]
Department of Comparative Medicine, University of Washington, Seattle, WA 98195, USA.
[4]
Department of Human Nutrition, Foods, and Exercise, Virginia Polytechnic Institute and State University, Blacksburg, VA 24061, USA.
[5]
Genethon, 91000 Evry, France.
[6]
Neuromuscular Physiology and Evaluation Lab, Institut de Myologie, 75651 Paris, France.
[7]
Division of Pediatric Pathology, Department of Pathology and Laboratory Medicine, Medical College of Wisconsin, Milwaukee, WI 53226, USA.
[8]
Department of Biomedical Engineering, University of Florida, Gainesville, FL 32611, USA.
[9]
Audentes Therapeutics, San Francisco, CA 94108, USA.
[10]
Department of Biomedical Informatics and Medical Education, University of Washington, Seattle, WA 98019, USA.
[11]
Department of Rehabilitation Medicine, University of Washington, Seattle, WA 98104, USA.
[12]
Department of Physical Therapy, University of Florida, Gainesville, FL 32610, USA.
[13]
Department of Medicine, Medical College of Wisconsin, Milwaukee, WI 53226, USA; Department of Cell Biology, Neurobiology and Anatomy, Medical College of Wisconsin, Milwaukee, WI 53226, USA; Cardiovascular Center, Medical College of Wisconsin, Milwaukee, WI 53226, USA.
[14]
The Manton Center for Orphan Disease Research, Boston Children's Hospital, Harvard Medical School, Boston, MA 02115, USA.
[15]
Genethon, 91000 Evry, France; INSERM, UMR_S951, 91002 Evry, France; Institut de Myologie, University Pierre and Marie Curie, 75005 Paris, France.
[16]
Genethon, 91000 Evry, France; INSERM, UMR_S951, 91002 Evry, France. Electronic address: abujbello@genethon.fr.
[17]
Department of Rehabilitation Medicine, University of Washington, Seattle, WA 98104, USA; Institute for Stem Cell and Regenerative Medicine, School of Medicine, University of Washington, Seattle, WA 98107, USA. Electronic address: mkc8@uw.edu.
[18]
主题词
动物(Animals);活组织检查(Biopsy);依赖病毒(Dependovirus);疾病模型, 动物(Disease Models, Animal);疾病恶化(Disease Progression);狗(Dogs);步态(Gait);基因表达(Gene Expression);遗传载体(Genetic Vectors);免疫, 细胞(Immunity, Cellular);免疫, 体液(Immunity, Humoral);Kaplan-Meiers评估(Kaplan-Meier Estimate);肌力(Muscle Strength);肌, 骨骼(Muscle, Skeletal);肌病, 结构性, 先天性(Myopathies, Structural, Congenital);蛋白质酪氨酸磷酸酶类, 非受体(Protein Tyrosine Phosphatases, Non-Receptor);功能恢复(Recovery of Function);反射(Reflex);呼吸功能试验(Respiratory Function Tests);组织分布(Tissue Distribution);转基因(Transgenes);治疗结果(Treatment Outcome)
DOI
10.1016/j.ymthe.2017.02.004
PMID
28237839
发布时间
2018-11-13
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