In Vivo Genome Editing Partially Restores Alpha1-Antitrypsin in a Murine Model of AAT Deficiency

Human Gene Therapy

Volumn 29, Issue 8, 2018, Pages 853-860

  Song, Chun Qing ^a   Wang, Dan ^a   Jiang, Tingting ^a   O'Connor, Kevin ^a   Tang, Qiushi ^a   Cai, Lingling ^b   Li, Xiangrui ^b   Weng, Zhiping ^a,b   Yin, Hao ^c   Gao, Guangping ^a   Mueller, Christian ^a   Flotte, Terence R ^a   Xue, Wen ^a  

^a UNIVERSITY OF MASSACHUSETTS   (United States)

^b TONGJI UNIVERSITY   (China)

^c WUHAN UNIVERSITY   (China)

Author keywords

AAT; AAV; alpha1 antitrypsin deficiency; CRISPR; genome editing; mouse model

Indexed keywords

ADENO ASSOCIATED VIRUS VECTOR; ALPHA 1 ANTITRYPSIN; GENOMIC DNA; GUIDE RNA; MYC PROTEIN; NUCLEOTIDE;

ADULT; ALPHA 1 ANTITRYPSIN DEFICIENCY; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; CRISPR-CAS9 SYSTEM; DNA REPAIR; ENZYME REPLACEMENT; EXPLORATORY RESEARCH; FEASIBILITY STUDY; GENE EDITING; GENE LOCUS; HOMOLOGY DIRECTED REPAIR; HUMAN; HUMAN CELL; IN VIVO STUDY; INDEL MUTATION; MOUSE; MUTANT; MUTATION RATE; NONHUMAN; NUCLEOTIDE SEQUENCE; POINT MUTATION; PROOF OF CONCEPT; VIRAL GENE DELIVERY SYSTEM; ANIMAL; CRISPR CAS SYSTEM; DEPENDOPARVOVIRUS; DISEASE MODEL; DRUG THERAPY; GENE THERAPY; GENE VECTOR; GENETICS; MUTATION; PATHOLOGY; TRANSGENIC MOUSE;

ALPHA 1-ANTITRYPSIN; ALPHA 1-ANTITRYPSIN DEFICIENCY; ANIMALS; CRISPR-CAS SYSTEMS; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; GENE EDITING; GENETIC THERAPY; GENETIC VECTORS; HUMANS; MICE; MICE, TRANSGENIC; MUTATION;

EID: 85050764363     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2017.225     Document Type: Article

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