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

Molecular Therapy

Volumn 25, Issue 4, 2017, Pages 839-854

  Mack, David L ^a   Poulard, Karine ^b,c   Goddard, Melissa A ^a   Latournerie, Virginie ^b,c   Snyder, Jessica M ^d   Grange, Robert W ^e   Elverman, Matthew R ^a   Denard, Jérôme ^b   Veron, Philippe ^b,c   Buscara, Laurine ^b,c   Le Bec, Christine ^b   Hogrel, Jean Yves ^f   Brezovec, Annie G ^e   Meng, Hui ^g   Yang, Lin ^h   Liu, Fujun ^h   O'Callaghan, Michael ⁱ   Gopal, Nikhil ^d   Kelly, Valerie E ^a   Smith, Barbara K ^j   Strande, Jennifer L ^g   Mavilio, Fulvio ^b,c   Beggs, Alan H ^k   Mingozzi, Federico ^b,c,l   Lawlor, Michael W ^g   Buj Bello, Ana ^b,c   Childers, Martin K ^a   more..

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

^b GÉNÉTHON   (France)

^c INSERM   (France)

^d UNIVERSITY OF WASHINGTON   (United States)

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

^f Institut de Myologie   (France)

^g MEDICAL COLLEGE OF WISCONSIN   (United States)

^h University of Florida   (United States)

ⁱ Audentes Therapeutics   (United States)

^j UNIVERSITY OF FLORIDA   (United States)

^k BOSTON CHILDREN S HOSPITAL   (United States)

^l SORBONNE UNIVERSITÉ   (France)

more..

Author keywords

adeno associated virus; canine; centronuclear; gene therapy; muscle; myopathy; myotubular; myotubularin; neuromuscular; pediatric

Indexed keywords

ADENO ASSOCIATED VIRUS VECTOR; MYOTUBULARIN; PHOSPHOINOSITIDE PHOSPHATASE; UNCLASSIFIED DRUG; NON RECEPTOR PROTEIN TYROSINE PHOSPHATASE;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CANINE MODEL; CENTRONUCLEAR MYOPATHY; CONTROLLED STUDY; DOSE RESPONSE; DRUG DOSE ESCALATION; DRUG EFFICACY; DRUG MEGADOSE; DRUG SAFETY; FEMALE; GAIT; GENE EXPRESSION; IMMUNE RESPONSE; LOW DRUG DOSE; MALE; MUSCLE STRENGTH; NERVOUS SYSTEM FUNCTION; NONHUMAN; ORGAN DISTRIBUTION; PEAK INSPIRATORY FLOW; SKELETAL MUSCLE; SPINAL REFLEX; SURVIVAL TIME; TISSUE DISTRIBUTION; VIRAL GENE THERAPY; ADMINISTRATION AND DOSAGE; ADVERSE EFFECTS; ANIMAL; BIOPSY; CELLULAR IMMUNITY; CLASSIFICATION; CONVALESCENCE; DEPENDOPARVOVIRUS; DISEASE COURSE; DISEASE MODEL; DOG; GENE THERAPY; GENE VECTOR; GENETICS; HUMORAL IMMUNITY; IMMUNOLOGY; KAPLAN MEIER METHOD; LUNG FUNCTION TEST; METABOLISM; MORTALITY; MYOPATHIES, STRUCTURAL, CONGENITAL; PATHOLOGY; PATHOPHYSIOLOGY; PHARMACOKINETICS; PROCEDURES; REFLEX; TRANSGENE; TREATMENT OUTCOME; ULTRASTRUCTURE;

ANIMALS; BIOPSY; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; DISEASE PROGRESSION; DOGS; GAIT; GENE EXPRESSION; GENETIC THERAPY; GENETIC VECTORS; IMMUNITY, CELLULAR; IMMUNITY, HUMORAL; KAPLAN-MEIER ESTIMATE; MUSCLE STRENGTH; MUSCLE, SKELETAL; MYOPATHIES, STRUCTURAL, CONGENITAL; PROTEIN TYROSINE PHOSPHATASES, NON-RECEPTOR; RECOVERY OF FUNCTION; REFLEX; RESPIRATORY FUNCTION TESTS; TISSUE DISTRIBUTION; TRANSGENES; TREATMENT OUTCOME;

EID: 85016950473     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1016/j.ymthe.2017.02.004     Document Type: Article

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