Gene therapy prolongs survival and restores function in murine and canine models of myotubular myopathy

Science Translational Medicine

Volumn 6, Issue 220, 2014, Pages

  Childers, Martin K ^a,b   Joubert, Romain ^c   Poulard, Karine ^c   Moal, Christelle ^c   Grange, Robert W ^d   Doering, Jonathan A ^d   Lawlor, Michael W ^e,f   Rider, Branden E ^e   Jamet, Thibaud ^c   Danièle, Nathalie ^c   Martin, Samia ^c   Rivière, Christel ^c   Soker, Thomas ^f   Hammer, Caroline ^c   Van Wittenberghe, Laetitia ^c   Lockard, Mandy ^g   Guan, Xuan ^g   Goddard, Melissa ^g   Mitchell, Erin ^g   Barber, Jane ^g   Williams, J Koudy ^g   Mack, David L ^a   Furth, Mark E ^h   Vignaud, Alban ^c   Masurier, Carole ^c   Mavilio, Fulvio ^c   Moullier, Philippe ^c,i,j   Beggs, Alan H ^e   Buj Bello, Anna ^c   more..

^a UNIVERSITY OF WASHINGTON SCHOOL OF MEDICINE   (United States)

^b UNIVERSITY OF WASHINGTON   (United States)

^c GÉNÉTHON   (France)

^d VIRGINIA POLYTECHNIC INSTITUTE AND STATE UNIVERSITY   (United States)

^e BOSTON CHILDREN S HOSPITAL   (United States)

^f MEDICAL COLLEGE OF WISCONSIN   (United States)

^g WAKE FOREST SCHOOL OF MEDICINE   (United States)

^h WAKE FOREST UNIVERSITY HEALTH SCIENCES   (United States)

ⁱ HÔTEL DIEU   (France)

^j UNIVERSITY OF FLORIDA   (United States)

more..

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR; AGENTS ACTING ON THE PERIPHERAL NERVOUS AND NEUROMUSCULAR SYSTEMS; MYOTUBULARIN; UNCLASSIFIED DRUG;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CENTRONUCLEAR MYOPATHY; CONTROLLED STUDY; DOG; DRUG EFFICACY; DRUG TOLERABILITY; IMMUNE RESPONSE; MALE; MOUSE; MUSCLE CONTRACTION; MUSCLE FUNCTION; MUSCLE WEAKNESS; NONHUMAN; PRIORITY JOURNAL; RESPIRATORY FAILURE; SURVIVAL TIME; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY;

ANIMALS; DEPENDOVIRUS; DIAPHRAGM; DISEASE MODELS, ANIMAL; DOGS; GENETIC THERAPY; GENETIC VECTORS; GENOTYPE; HEK293 CELLS; HUMANS; MALE; MICE; MICE, KNOCKOUT; MUSCLE CONTRACTION; MUSCLE WEAKNESS; MUTATION; MYOPATHIES, STRUCTURAL, CONGENITAL; PROTEIN TYROSINE PHOSPHATASES, NON-RECEPTOR;

EID: 84893427190     PISSN: 19466234     EISSN: 19466242     Source Type: Journal    
DOI: 10.1126/scitranslmed.3007523     Document Type: Article

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