Selection-free gene repair after adenoviral vector transduction of designer nucleases: Rescue of dystrophin synthesis in DMD muscle cell populations

Nucleic Acids Research

Volumn 44, Issue 3, 2016, Pages 1449-1470

  Maggio, Ignazio ^a   Stefanucci, Luca ^a,b   Janssen, Josephine M ^a   Liu, Jin ^a   Chen, Xiaoyu ^a   Mouly, Vincent ^c   Gonçalves, Manuel A F V ^a  

^a LEIDEN UNIVERSITY MEDICAL CENTER   (Netherlands)

^b UNIVERSITY OF ROME TOR VERGATA   (Italy)

^c CNRS   (France)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR; DYSTROPHIN; MESSENGER RNA; NUCLEASE; RNA GUIDED NUCLEASE; TRANSCRIPTIONAL ACTIVATOR LIKE EFFECTOR NUCLEASE; UNCLASSIFIED DRUG; ENDONUCLEASE; GUIDE RNA;

ALLELE; ARTICLE; CONTROLLED STUDY; CRISPR CAS SYSTEM; DMD GENE; DNA END JOINING REPAIR; DNA SEQUENCE; DUCHENNE MUSCULAR DYSTROPHY; EXON SKIPPING; FLOW CYTOMETRY; GENE; GENE DELETION; GENE INSERTION; GENE MUTATION; GENETIC TRANSDUCTION; GENOTYPE; HEK293 CELL LINE; HELA CELL LINE; HUMAN; HUMAN CELL; IMMUNOFLUORESCENCE MICROSCOPY; MUSCLE CELL; MUSCLE STEM CELL; MYOBLAST; PLASMID; PRIORITY JOURNAL; READING FRAME; WESTERN BLOTTING; ADENOVIRIDAE; CELL LINE; FLUORESCENCE MICROSCOPY; GENE THERAPY; GENE VECTOR; GENETICS; METABOLISM; MOLECULAR GENETICS; MUSCULAR DYSTROPHY, DUCHENNE; MUTATION; NUCLEOTIDE SEQUENCE; PROCEDURES;

ADENOVIRIDAE; ALLELES; BASE SEQUENCE; BLOTTING, WESTERN; CELL LINE; CRISPR-CAS SYSTEMS; DNA END-JOINING REPAIR; DYSTROPHIN; ENDONUCLEASES; GENETIC THERAPY; GENETIC VECTORS; HEK293 CELLS; HELA CELLS; HUMANS; MICROSCOPY, FLUORESCENCE; MOLECULAR SEQUENCE DATA; MUSCULAR DYSTROPHY, DUCHENNE; MUTATION; MYOBLASTS; RNA, GUIDE; TRANSDUCTION, GENETIC;

EID: 84965050620     PISSN: 03051048     EISSN: 13624962     Source Type: Journal    
DOI: 10.1093/nar/gkv1540     Document Type: Article

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