CRISPR/Cas9 Editing of the Mutant Huntingtin Allele In Vitro and In Vivo

Molecular Therapy

Volumn 25, Issue 1, 2017, Pages 12-23

  Monteys, Alex Mas ^a   Ebanks, Shauna A ^a   Keiser, Megan S ^a   Davidson, Beverly L ^a,b  

^a CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

^b UNIVERSITY OF PENNSYLVANIA   (United States)

Author keywords

AAV; CRISPR Cas9; gene therapy; genome editing; Huntington's disease

Indexed keywords

GENOMIC DNA; HUNTINGTIN; MESSENGER RNA; RNA; SINGLE GUIDE RNA; UNCLASSIFIED DRUG; BACTERIAL PROTEIN; CAS9 ENDONUCLEASE STREPTOCOCCUS PYOGENES; ENDONUCLEASE; GUIDE RNA;

ALLELE; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CLUSTERED REGULARLY INTERSPACED SHORT PALINDROMIC REPEAT; CONTROLLED STUDY; CRISPR CAS9 SYSTEM; DNA STRUCTURE; GENE EDITING; GENE EXPRESSION REGULATION; GENE LOCUS; GENETIC REGULATION; HEK293 CELL LINE; HETEROZYGOSITY; HUMAN; HUMAN CELL; HUNTINGTON CHOREA; IN VITRO STUDY; IN VIVO STUDY; MALE; MOLECULAR PATHOLOGY; MOUSE; NONHUMAN; PROMOTER REGION; PROTOSPACER ADJACENT MOTIF; SINGLE NUCLEOTIDE POLYMORPHISM; ANIMAL; CRISPR CAS SYSTEM; DISEASE MODEL; EXON; FIBROBLAST; GENE ORDER; GENE SILENCING; GENETICS; METABOLISM; MUTATION; NUCLEOTIDE MOTIF; PLASMID; TRANSGENIC MOUSE;

ALLELES; ANIMALS; BACTERIAL PROTEINS; CLUSTERED REGULARLY INTERSPACED SHORT PALINDROMIC REPEATS; CRISPR-CAS SYSTEMS; DISEASE MODELS, ANIMAL; ENDONUCLEASES; EXONS; FIBROBLASTS; GENE EDITING; GENE ORDER; GENE SILENCING; HUMANS; HUNTINGTIN PROTEIN; HUNTINGTON DISEASE; MICE; MICE, TRANSGENIC; MUTATION; NUCLEOTIDE MOTIFS; PLASMIDS; POLYMORPHISM, SINGLE NUCLEOTIDE; PROMOTER REGIONS, GENETIC; RNA, GUIDE;

EID: 85016289126     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1016/j.ymthe.2016.11.010     Document Type: Article

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