In vivo genome editing restores dystrophin expression and cardiac function in dystrophic mice

Circulation Research

Volumn 121, Issue 8, 2017, Pages 923-929

  El Refaey, Mona ^a   Xu, Li ^a   Gao, Yandi ^a   Canan, Benjamin D ^a   Ayodele Adesanya, T M ^a   Warner, Sarah C ^b   Akagi, Keiko ^c   Symer, David E ^b,c   Mohler, Peter J ^a   Ma, Jianjie ^a   Janssen, Paul M L ^d   Han, Renzhi ^a  

^a OHIO STATE UNIVERSITY   (United States)

^b OHIO STATE UNIVERSITY   (United States)

^c OHIO STATE UNIVERSITY   (United States)

^d NONE

Author keywords

Cardiomyopathies; Dystrophin; Gene editing; Mice; Muscular dystrophy

Indexed keywords

ADENO ASSOCIATED VIRUS VECTOR; DYSTROPHIN; GENOMIC DNA; GUIDE RNA; APO-DYSTROPHIN 1; CRISPR ASSOCIATED PROTEIN; UTROPHIN;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CARDIOMYOPATHY; CONTROLLED STUDY; CRISPR-CAS9 SYSTEM; DNA END JOINING REPAIR; DUCHENNE MUSCULAR DYSTROPHY; GENE EDITING; HEART MUSCLE CONTRACTILITY; HEART MUSCLE FIBROSIS; HEART PAPILLARY MUSCLE; MOUSE; NEWBORN; NONHUMAN; POINT MUTATION; PRIORITY JOURNAL; PROTEIN EXPRESSION; STAPHYLOCOCCUS AUREUS; STREPTOCOCCUS PYOGENES; X CHROMOSOME LINKED MUSCULAR DYSTROPHIC MOUSE; ANIMAL; CARDIOMYOPATHIES; CLUSTERED REGULARLY INTERSPACED SHORT PALINDROMIC REPEAT; CONVALESCENCE; CRISPR CAS SYSTEM; DEPENDOPARVOVIRUS; DISEASE MODEL; EXON; FIBROSIS; GENE EXPRESSION REGULATION; GENE THERAPY; GENE VECTOR; GENETIC PREDISPOSITION; GENETICS; HEART CONTRACTION; HIGH THROUGHPUT SEQUENCING; METABOLISM; MUSCULAR DYSTROPHY, DUCHENNE; MUTATION; PATHOLOGY; PATHOPHYSIOLOGY; PHENOTYPE; PROCEDURES;

ANIMALS; CARDIOMYOPATHIES; CLUSTERED REGULARLY INTERSPACED SHORT PALINDROMIC REPEATS; CRISPR-ASSOCIATED PROTEINS; CRISPR-CAS SYSTEMS; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; DYSTROPHIN; EXONS; FIBROSIS; GENE EDITING; GENE EXPRESSION REGULATION; GENETIC PREDISPOSITION TO DISEASE; GENETIC THERAPY; GENETIC VECTORS; HIGH-THROUGHPUT NUCLEOTIDE SEQUENCING; MICE, INBRED MDX; MUSCULAR DYSTROPHY, DUCHENNE; MUTATION; MYOCARDIAL CONTRACTION; PAPILLARY MUSCLES; PHENOTYPE; RECOVERY OF FUNCTION; RNA, GUIDE; UTROPHIN;

EID: 85031102132     PISSN: 00097330     EISSN: 15244571     Source Type: Journal    
DOI: 10.1161/CIRCRESAHA.117.310996     Document Type: Article

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