Cas9/gRNA targeted excision of cystic fibrosis-causing deep-intronic splicing mutations restores normal splicing of CFTR mRNA

PLoS ONE

Volumn 12, Issue 9, 2017, Pages

  Sanz, David J ^a   Hollywood, Jennifer A ^a,b   Scallan, Martina F ^a   Harrison, Patrick T ^a  

^a UNIVERSITY COLLEGE CORK   (Ireland)

^b UNIVERSITY OF AUCKLAND   (New Zealand)

Author keywords

[No Author keywords available]

Indexed keywords

ARTICLE; CFTR GENE; CONTROLLED STUDY; CRISPR-CAS9 SYSTEM; CYSTIC FIBROSIS; DNA END JOINING REPAIR; DOUBLE STRANDED DNA BREAK; FLUORESCENCE ACTIVATED CELL SORTING; GENE; GENE EDITING; GENE MUTATION; GENETIC ANALYSIS; GENETIC VARIABILITY; HUMAN; HUMAN CELL; MOLECULAR CLONING; MUTATIONAL ANALYSIS; RNA SPLICING; TRANSCRIPTION REGULATION; ALTERNATIVE RNA SPLICING; CELL SEPARATION; CODON; CRISPR CAS SYSTEM; EXON; FLOW CYTOMETRY; GENE DELETION; GENETICS; INTRON; METABOLISM; MUTATION; PROTEIN MOTIF;

CFTR PROTEIN, HUMAN; CODON; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; GUIDE RNA; MESSENGER RNA; OLIGONUCLEOTIDE;

ALTERNATIVE SPLICING; AMINO ACID MOTIFS; CELL SEPARATION; CODON; CRISPR-CAS SYSTEMS; CYSTIC FIBROSIS; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; EXONS; FLOW CYTOMETRY; GENE DELETION; HUMANS; INTRONS; MUTATION; OLIGONUCLEOTIDES; RNA, GUIDE; RNA, MESSENGER;

EID: 85029890340     PISSN: None     EISSN: 19326203     Source Type: Journal    
DOI: 10.1371/journal.pone.0184009     Document Type: Article

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