Correction of a splicing defect in a mouse model of congenital muscular dystrophy type 1A using a homology-directed-repair-independent mechanism

Nature Medicine

Volumn 23, Issue 8, 2017, Pages 984-989

  Kemaladewi, Dwi U ^a   Maino, Eleonora ^a,b   Hyatt, Elzbieta ^a   Hou, Huayun ^a,b   Ding, Maylynn ^a   Place, Kara M ^a   Zhu, Xinyi ^a   Bassi, Prabhpreet ^a,b   Baghestani, Zahra ^a   Deshwar, Amit G ^c   Merico, Daniele ^a,c,d   Xiong, Hui Y ^c   Frey, Brendan J ^c,e   Wilson, Michael D ^a,b,f   Ivakine, Evgueni A ^a   Cohn, Ronald D ^a,b,g  

^a HOSPITAL FOR SICK CHILDREN RESEARCH INSTITUTE   (Canada)

^b UNIVERSITY OF TORONTO   (Canada)

^c Deep Genomics Inc   (Canada)

^d HOSPITAL FOR SICK CHILDREN   (Canada)

^e UNIVERSITY OF TORONTO   (Canada)

^f HEART AND STROKE FOUNDATION OF CANADA   (Canada)

^g HOSPITAL FOR SICK CHILDREN   (Canada)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR; LAMININ ALPHA2; MESSENGER RNA; LAMININ; RNA SPLICE SITE;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CONGENITAL MUSCULAR DYSTROPHY TYPE 1A; CONTROLLED STUDY; CRISPR-CAS9 SYSTEM; DNA END JOINING REPAIR; GENE EDITING; HISTOPATHOLOGY; INTRON; MOUSE; MOUSE MODEL; MUSCLE ATROPHY; MUSCULAR DYSTROPHY; NEWBORN; NONHUMAN; PARALYSIS; PATHOGENESIS; POINT MUTATION; PRIORITY JOURNAL; SPLICING DEFECT; ANIMAL; CRISPR CAS SYSTEM; DISEASE MODEL; FLUORESCENT ANTIBODY TECHNIQUE; GENE THERAPY; GENETICS; METABOLISM; MUTATION; PATHOLOGY; PROCEDURES; REAL TIME POLYMERASE CHAIN REACTION; RNA SPLICE SITE; SKELETAL MUSCLE; WESTERN BLOTTING;

ANIMALS; BLOTTING, WESTERN; CRISPR-CAS SYSTEMS; DISEASE MODELS, ANIMAL; DNA END-JOINING REPAIR; FLUORESCENT ANTIBODY TECHNIQUE; GENETIC THERAPY; LAMININ; MICE; MUSCLE, SKELETAL; MUSCULAR DYSTROPHIES; MUTATION; REAL-TIME POLYMERASE CHAIN REACTION; RNA SPLICE SITES; RNA, MESSENGER;

EID: 85029315887     PISSN: 10788956     EISSN: 1546170X     Source Type: Journal    
DOI: 10.1038/nm.4367     Document Type: Article

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