Efficient and allele-specific genome editing of disease loci in human iPSCs

Molecular Therapy

Volumn 23, Issue 3, 2015, Pages 570-577

  Smith, Cory ^a   Abalde Atristain, Leire ^a   He, Chaoxia ^a   Brodsky, Brett R ^a   Braunstein, Evan M ^a   Chaudhari, Pooja ^a,b   Jang, Yoon Young ^a,b   Cheng, Linzhao ^a   Ye, Zhaohui ^a  

^a JOHNS HOPKINS UNIVERSITY SCHOOL OF MEDICINE   (United States)

^b JOHNS HOPKINS UNIVERSITY   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ALPHA 1 ANTITRYPSIN; CRISPR ASSOCIATED PROTEIN; JANUS KINASE 2; NUCLEOTIDE; PHENYLALANINE; TRANSCRIPTION ACTIVATOR LIKE EFFECTOR NUCLEASE; VALINE; BACTERIAL PROTEIN; CAS9 PROTEIN, FRANCISELLA NOVICIDA; ENDONUCLEASE; GUIDE RNA;

ALLELE; ARTICLE; CONTROLLED STUDY; DNA END JOINING REPAIR; GENE DELETION; GENE DISRUPTION; GENE FREQUENCY; GENE INSERTION; GENE LOCUS; GENE TARGETING; HETEROZYGOTE; HUMAN; HUMAN CELL; HUMAN GENOME; INHERITANCE; LIVING DONOR; MUTANT; MYELOPROLIFERATIVE NEOPLASM; PLURIPOTENT STEM CELL; POINT MUTATION; PROTEIN DEPLETION; PROTEIN EXPRESSION; PROTEIN RNA BINDING; RNA EDITING; CELL LINE; CLUSTERED REGULARLY INTERSPACED SHORT PALINDROMIC REPEAT; GENE EXPRESSION; GENETICS; INDUCED PLURIPOTENT STEM CELL; METABOLISM; MOLECULAR GENETICS; MUTATION; MYELOPROLIFERATIVE DISORDER; NUCLEOTIDE SEQUENCE; PATHOLOGY; QUANTITATIVE TRAIT LOCUS; RECOMBINATION REPAIR;

ALLELES; ALPHA 1-ANTITRYPSIN; BACTERIAL PROTEINS; BASE SEQUENCE; CELL LINE; CLUSTERED REGULARLY INTERSPACED SHORT PALINDROMIC REPEATS; DNA END-JOINING REPAIR; ENDONUCLEASES; GENE EXPRESSION; GENOME, HUMAN; HUMANS; INDUCED PLURIPOTENT STEM CELLS; JANUS KINASE 2; MOLECULAR SEQUENCE DATA; MUTATION; MYELOPROLIFERATIVE DISORDERS; QUANTITATIVE TRAIT LOCI; RECOMBINATIONAL DNA REPAIR; RNA, GUIDE;

EID: 84924033062     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2014.226     Document Type: Article

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