Permanent inactivation of Huntington's disease mutation by personalized allele-specific CRISPR/Cas9

Human Molecular Genetics

Volumn 25, Issue 20, 2016, Pages 4566-4576

  Shin, Jun Wan ^a,b   Kim, Kyung Hee ^a,b   Chao, Michael J ^a,b   Atwal, Ranjit S ^a,b   Gillis, Tammy ^a   MacDonald, Marcy E ^a,b,c   Gusella, James F ^a,b,c   Lee, Jong Min ^a,b,c  

^a MASSACHUSETTS GENERAL HOSPITAL   (United States)

^b HARVARD MEDICAL SCHOOL   (United States)

^c BROAD INSTITUTE OF MIT AND HARVARD   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

GUIDE RNA; MESSENGER RNA; HTT PROTEIN, HUMAN; HUNTINGTIN;

ARTICLE; CAG REPEAT; CHROMOSOME; CLUSTERED REGULARLY INTERSPACED SHORT PALINDROMIC REPEAT; CONTROLLED STUDY; DNA STRUCTURE; GAIN OF FUNCTION MUTATION; GENE INACTIVATION; GENETIC VARIATION; GENOTYPE; HAPLOTYPE; HUMAN; HUNTINGTON CHOREA; PERSONALIZED MEDICINE; POINT MUTATION; PRIORITY JOURNAL; PROMOTER REGION; SANGER SEQUENCING; SINGLE NUCLEOTIDE POLYMORPHISM; TRANSCRIPTION INITIATION SITE; WESTERN BLOTTING; CELL LINE; CRISPR CAS SYSTEM; FIBROBLAST; GENE THERAPY; GENETICS; MALE; METABOLISM; MIDDLE AGED; PROCEDURES; TRINUCLEOTIDE REPEAT;

CELL LINE; CRISPR-CAS SYSTEMS; FIBROBLASTS; GENETIC THERAPY; HAPLOTYPES; HUMANS; HUNTINGTIN PROTEIN; HUNTINGTON DISEASE; MALE; MIDDLE AGED; POLYMORPHISM, SINGLE NUCLEOTIDE; PRECISION MEDICINE; TRINUCLEOTIDE REPEAT EXPANSION;

EID: 85007221153     PISSN: 09646906     EISSN: 14602083     Source Type: Journal    
DOI: 10.1093/hmg/ddw286     Document Type: Article

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