Gene therapy in monogenic congenital myopathies

Methods

Volumn 99, Issue , 2016, Pages 91-98

  Guan, Xuan ^a,b,c   Goddard, Melissa A ^a,b,c   Mack, David L ^b,c   Childers, Martin K ^b,c  

^a WAKE FOREST UNIVERSITY HEALTH SCIENCES   (United States)

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

^c UNIVERSITY OF WASHINGTON   (United States)

Author keywords

Animal models; Gene therapy; Muscle disease; Muscular dystrophy; Myopathy

Indexed keywords

ADENOVIRUS VECTOR; ANTISENSE OLIGONUCLEOTIDE; MORPHOLINO OLIGONUCLEOTIDE; VIRUS VECTOR; MUSCLE PROTEIN;

CELL CULTURE; CENTRONUCLEAR MYOPATHY; CLINICAL EFFECTIVENESS; CLINICAL TRIAL (TOPIC); CONGENITAL DISORDER; DISEASE MODEL; GENE DELIVERY SYSTEM; GENE MUTATION; GENE REPLACEMENT THERAPY; GENE VECTOR; HUMAN; INJECTION; INTERMETHOD COMPARISON; MONOGENIC CONGENITAL MYOPATHY; MYOPATHY; NONHUMAN; PRIORITY JOURNAL; REVIEW; TREATMENT INDICATION; ANIMAL; GENE THERAPY; GENE TRANSFER; GENETICS; MYOPATHIES, STRUCTURAL, CONGENITAL;

ANIMALS; GENE TRANSFER TECHNIQUES; GENETIC THERAPY; HUMANS; MUSCLE PROTEINS; MYOPATHIES, STRUCTURAL, CONGENITAL;

EID: 84951818948     PISSN: 10462023     EISSN: 10959130     Source Type: Journal    
DOI: 10.1016/j.ymeth.2015.10.004     Document Type: Review

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