Enzyme replacement therapy rescues weakness and improves muscle pathology in mice with X-linked myotubular myopathy

Human Molecular Genetics

Volumn 22, Issue 8, 2013, Pages 1525-1538

  Lawlor, Michael W ^a,b   Armstrong, Dustin ^c   Viola, Marissa G ^a   Widrick, Jeffrey J ^a,d   Meng, Hui ^b   Grange, Robert W ^e   Childers, Martin K ^f   Hsu, Cynthia P ^a   O'Callaghan, Michael ^c   Pierson, Christopher R ^g   Buj Bello, Anna ^h   Beggs, Alan H ^a  

^a BOSTON CHILDREN S HOSPITAL   (United States)

^b MEDICAL COLLEGE OF WISCONSIN   (United States)

^c 4s3 Bioscience Inc   (United States)

^d and Red Sox Foundation and Massachusetts General Hospital Home Base Program   (United States)

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

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

^g NATIONWIDE CHILDREN S HOSPITAL   (United States)

^h GÉNÉTHON   (France)

Author keywords

[No Author keywords available]

Indexed keywords

3E10FV MTM1; MYOTUBULARIN; PHOSPHATASE; RECOMBINANT ENZYME; UNCLASSIFIED DRUG;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CENTRONUCLEAR MYOPATHY; CONTROLLED STUDY; ENZYME DEFICIENCY; ENZYME REPLACEMENT; EXTENSOR DIGITORUM LONGUS MUSCLE; IMMUNOFLUORESCENCE; IMMUNOHISTOCHEMISTRY; LOW DRUG DOSE; MALE; MOUSE; MUSCLE CELL; MUSCLE CONTRACTION; MUSCLE WEAKNESS; NONHUMAN; PHENOTYPE; PRIORITY JOURNAL; QUADRICEPS FEMORIS MUSCLE; SOLEUS MUSCLE; TIBIALIS ANTERIOR MUSCLE; WILD TYPE; X LINKED MYOTUBULAR MYOPATHY;

ANIMALS; DISEASE MODELS, ANIMAL; ENZYME REPLACEMENT THERAPY; FATIGUE; FEMALE; HUMANS; MICE; MUSCLE WEAKNESS; MUSCLE, SKELETAL; MUSCLES; MYOPATHIES, STRUCTURAL, CONGENITAL; PROTEIN TYROSINE PHOSPHATASES, NON-RECEPTOR;

MUS;

EID: 84875766052     PISSN: 09646906     EISSN: 14602083     Source Type: Journal    
DOI: 10.1093/hmg/ddt003     Document Type: Article

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