Correction of diverse muscular dystrophy mutations in human engineered heart muscle by single-site genome editing

Science Advances

Volumn 4, Issue 1, 2018, Pages

  Long, Chengzu ^a,b   Li, Hui ^a   Tiburcy, Malte ^c,d   Rodriguez Caycedo, Cristina ^a   Kyrychenko, Viktoriia ^a   Zhou, Huanyu ^a   Zhang, Yu ^a   Min, Yi Li ^a   Shelton, John M ^e   Mammen, Pradeep P A ^a,e   Liaw, Norman Y ^c   Zimmermann, Wolfram Hubertus ^c,d   Bassel Duby, Rhonda ^a   Schneider, Jay W ^a,e   Olson, Eric N ^a  

^a UNIVERSITY OF TEXAS SOUTHWESTERN MEDICAL CENTER   (United States)

^b NEW YORK UNIVERSITY SCHOOL OF MEDICINE   (United States)

^c UNIVERSITY OF GÖTTINGEN   (Germany)

^d DZHK GERMAN CENTRE FOR CARDIOVASCULAR RESEARCH   (Germany)

^e UNIVERSITY OF TEXAS SOUTHWESTERN MEDICAL CENTER   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

GENE EXPRESSION; HEART; MACHINERY; MUSCLE; NUCLEIC ACIDS; RESTORATION; RNA; STEM CELLS;

DUCHENNE MUSCULAR DYSTROPHIES; INDUCED PLURIPOTENT STEM CELLS; MECHANICAL FORCE; MUSCULAR DYSTROPHY; NONHOMOLOGOUS END JOINING; PROOF OF CONCEPT; PROTEIN EXPRESSIONS; SPLICING MACHINERY;

GENES;

DYSTROPHIN; GUIDE RNA; MESSENGER RNA;

CARDIAC MUSCLE; CARDIAC MUSCLE CELL; DUCHENNE MUSCULAR DYSTROPHY; EXON; GENE EDITING; GENETICS; HUMAN; HUMAN GENOME; INDUCED PLURIPOTENT STEM CELL; METABOLISM; MUTATION; NUCLEOTIDE SEQUENCE; PATHOLOGY; PROCEDURES; TISSUE ENGINEERING;

BASE SEQUENCE; DYSTROPHIN; EXONS; GENE EDITING; GENOME, HUMAN; HUMANS; INDUCED PLURIPOTENT STEM CELLS; MUSCULAR DYSTROPHY, DUCHENNE; MUTATION; MYOCARDIUM; MYOCYTES, CARDIAC; RNA, GUIDE; RNA, MESSENGER; TISSUE ENGINEERING;

EID: 85042163565     PISSN: None     EISSN: 23752548     Source Type: Journal    
DOI: 10.1126/sciadv.aap9004     Document Type: Article

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