Survival Advantage of Both Human Hepatocyte Xenografts and Genome-Edited Hepatocytes for Treatment of α-1 Antitrypsin Deficiency

Molecular Therapy

Volumn 25, Issue 11, 2017, Pages 2477-2489

  Borel, Florie ^a   Tang, Qiushi ^a   Gernoux, Gwladys ^a   Greer, Cynthia ^a   Wang, Ziqiong ^a   Barzel, Adi ^b,c,d   Kay, Mark A ^d   Shultz, Leonard D ^e   Greiner, Dale L ^a   Flotte, Terence R ^a   Brehm, Michael A ^a   Mueller, Christian ^a  

^a UNIVERSITY OF MASSACHUSETTS MEDICAL SCHOOL   (United States)

^b Millennium BioTherapeutics Inc   (United States)

^c TEL AVIV UNIVERSITY   (Israel)

^d STANFORD UNIVERSITY SCHOOL OF MEDICINE   (United States)

^e JACKSON LABORATORY   (United States)

Author keywords

A1AT; AAT; AATD; AAV; gene editing; humanized liver mouse model; liver regeneration; liver xenograft; miRNA; nuclease free genome editing; RNAi; shRNA; 1 antitrypsin deficiency

Indexed keywords

ADENO ASSOCIATED VIRUS VECTOR; ALBUMIN; ALPHA 1 ANTITRYPSIN; MICRORNA; MUTANT PROTEIN; PIZ PROTEIN; UNCLASSIFIED DRUG;

ALLELE; ALPHA 1 ANTITRYPSIN DEFICIENCY; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CELL SURVIVAL; CELL TRANSFORMATION; CELL TRANSPLANTATION; CONTROLLED STUDY; ENGRAFTMENT; ENZYME SYNTHESIS; GENE EDITING; GENE SILENCING; GENE THERAPY; HUMAN; HUMAN CELL; IMMUNE DEFICIENCY; LIVER CELL DAMAGE; MOUSE; NONHUMAN; PROMOTER REGION; REPRODUCIBILITY; XENOGRAFT; XENOTRANSPLANTATION; ANIMAL; CHEMISTRY; DEPENDOPARVOVIRUS; DISEASE MODEL; ENZYMOLOGY; GENE EXPRESSION; GENE VECTOR; GENETICS; GRAFT SURVIVAL; LIVER CELL; MALE; METABOLISM; MUTATION; NONOBESE DIABETIC MOUSE; PATHOLOGY; PROCEDURES; TRANSGENE; TRANSPLANTATION;

ALPHA 1-ANTITRYPSIN; ALPHA 1-ANTITRYPSIN DEFICIENCY; ANIMALS; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; GENE EDITING; GENE EXPRESSION; GENE SILENCING; GENETIC THERAPY; GENETIC VECTORS; GRAFT SURVIVAL; HEPATOCYTES; HUMANS; MALE; MICE; MICE, INBRED NOD; MICRORNAS; MUTATION; TRANSGENES; TRANSPLANTATION, HETEROLOGOUS;

EID: 85030830011     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1016/j.ymthe.2017.09.020     Document Type: Article

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