Dual AAV Gene Therapy for Duchenne Muscular Dystrophy with a 7-kb Mini-Dystrophin Gene in the Canine Model

Human Gene Therapy

Volumn 29, Issue 3, 2018, Pages 299-311

  Kodippili, Kasun ^a   Hakim, Chady H ^a,b   Pan, Xiufang ^a   Yang, Hsiao T ^a,c   Yue, Yongping ^a   Zhang, Yadong ^a,e   Shin, Jin Hong ^a,f   Yang, N Nora ^b   Duan, Dongsheng ^a,c,d  

^a University of Missouri   (United States)

^b NATIONAL INSTITUTES OF HEALTH   (United States)

^c UNIVERSITY OF MISSOURI   (United States)

^d UNIVERSITY OF MISSOURI   (United States)

^e HUAZHONG UNIVERSITY OF SCIENCE AND TECHNOLOGY   (China)

^f Yangsan Hospital   (South Korea)

Author keywords

adeno associated virus; canine model; dual AAV vectors; Duchenne muscular dystrophy; dystrophin; mini dystrophin

Indexed keywords

ADENO ASSOCIATED VIRUS VECTOR; CYCLOSPORINE; DYSTROPHIN; DYSTROPHIN ASSOCIATED PROTEIN COMPLEX; MYCOPHENOLATE MOFETIL; DMD PROTEIN, HUMAN;

AMINO TERMINAL SEQUENCE; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CONTROLLED STUDY; DUCHENNE MUSCULAR DYSTROPHY; ECCENTRIC MUSCLE CONTRACTION; GENE EXPRESSION; GENE TRANSFER; GENETIC ENGINEERING; LIMIT OF QUANTITATION; MOUSE; MUSCLE ATROPHY; MUSCLE FUNCTION; NONHUMAN; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; ANIMAL; CONVALESCENCE; DEPENDOPARVOVIRUS; DISEASE MODEL; DOG; GENE THERAPY; GENETICS; HUMAN; METABOLISM; MUSCLE CONTRACTION; PATHOPHYSIOLOGY; PROCEDURES; SKELETAL MUSCLE;

ANIMALS; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; DOGS; DYSTROPHIN; GENETIC THERAPY; HUMANS; MUSCLE CONTRACTION; MUSCLE, SKELETAL; MUSCULAR DYSTROPHY, DUCHENNE; RECOVERY OF FUNCTION;

EID: 85043535458     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2017.095     Document Type: Article

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