PIK3C2B inhibition improves function and prolongs survival in myotubular myopathy animal models

Journal of Clinical Investigation

Volumn 126, Issue 9, 2016, Pages 3613-3625

  Sabha, Nesrin ^a   Volpatti, Jonathan R ^a,b   Gonorazky, Hernan ^a   Reifler, Aaron ^c   Davidson, Ann E ^a   Li, Xingli ^c   Eltayeb, Nadine M ^a   Dall'Armi, Claudia ^d   Di Paolo, Gilbert ^d   Brooks, Susan V ^c   Buj Bello, Ana ^e,f   Feldman, Eva L ^c   Dowling, James J ^a,b  

^a HOSPITAL FOR SICK CHILDREN   (Canada)

^b UNIVERSITY OF TORONTO   (Canada)

^c UNIVERSITY OF MICHIGAN MEDICAL SCHOOL   (United States)

^d New York Presbyterian Morgan Stanley Children's Hospital   (United States)

^e GÉNÉTHON   (France)

^f U951 ^*  (France)

Author keywords

[No Author keywords available]

Indexed keywords

2 (4 HYDROXYPHENYL) 4 MORPHOLINOPYRIDO[3',2':4,5]FURO[3,2 D]PYRIMIDINE; 2 MORPHOLINO 8 PHENYLCHROMONE; PHOSPHATIDYLINOSITOL 3 KINASE; PHOSPHATIDYLINOSITOL 3 KINASE C2B; UNCLASSIFIED DRUG; WORTMANNIN; ANDROSTANE DERIVATIVE; MYOTUBULARIN; NON RECEPTOR PROTEIN TYROSINE PHOSPHATASE; PHOSPHATIDYLINOSITOL 4 PHOSPHATE 3 KINASE; PIK3C2B PROTEIN, MOUSE; PIK3C3 PROTEIN, MOUSE;

ALLELE; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; AUTOPHAGY; CELLULAR DISTRIBUTION; CENTRONUCLEAR MYOPATHY; CONTROLLED STUDY; ENZYME INHIBITION; ENZYME LINKED IMMUNOSORBENT ASSAY; FEMALE; GENE INTERACTION; HAPLOINSUFFICIENCY; IMMUNOHISTOCHEMISTRY; MALE; MOTOR PERFORMANCE; MOUSE; NONHUMAN; PHENOTYPE; PRIORITY JOURNAL; PROTEIN EXPRESSION; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION; SKELETAL MUSCLE; SURVIVAL; WESTERN BLOTTING; ZEBRA FISH; ANIMAL; CHEMISTRY; DISEASE MODEL; DRUG EFFECTS; GENETICS; KNOCKOUT MOUSE; METABOLISM; MYOPATHIES, STRUCTURAL, CONGENITAL; PHYSIOLOGY; TRANSGENIC ANIMAL;

ANDROSTADIENES; ANIMALS; ANIMALS, GENETICALLY MODIFIED; CLASS II PHOSPHATIDYLINOSITOL 3-KINASES; DISEASE MODELS, ANIMAL; FEMALE; MALE; MICE; MICE, KNOCKOUT; MOTOR SKILLS; MUSCLE, SKELETAL; MYOPATHIES, STRUCTURAL, CONGENITAL; PHENOTYPE; PHOSPHATIDYLINOSITOL 3-KINASES; PROTEIN TYROSINE PHOSPHATASES, NON-RECEPTOR; ZEBRAFISH;

EID: 84987847671     PISSN: 00219738     EISSN: 15588238     Source Type: Journal    
DOI: 10.1172/JCI86841     Document Type: Article

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