Curative ex vivo liver-directed gene therapy in a pig model of hereditary tyrosinemia type 1

Science Translational Medicine

Volumn 8, Issue 349, 2016, Pages

  Hickey, Raymond D ^a   Mao, Shennen A ^a   Glorioso, Jaime ^a   Elgilani, Faysal ^a   Amiot, Bruce ^b   Chen, Harvey ^a   Rinaldo, Piero ^a   Marler, Ronald ^c   Jiang, Huailei ^a   DeGrado, Timothy R ^a   Suksanpaisan, Lukkana ^a,d   O'Connor, Michael K ^a   Freeman, Brittany L ^a   Ibrahim, Samar H ^a   Peng, Kah Whye ^a   Harding, Cary O ^e   Ho, Chak Sum ^f   Grompe, Markus ^e   Ikeda, Yasuhiro ^a   Lillegard, Joseph B ^a,g   Russell, Stephen J ^a   Nyberg, Scott L ^a   more..

^a MAYO CLINIC   (United States)

^b Brami Biomedical Inc   (United States)

^c MAYO CLINIC   (United States)

^d Imanis Life Sciences   (United States)

^e OREGON HEALTH AND SCIENCE UNIVERSITY   (United States)

^f Histocompatibility Laboratory   (United States)

^g CHILDREN S HOSPITALS AND CLINICS OF MINNESOTA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

COLLAGENASE; FUMARYLACETOACETASE; LENTIVIRUS VECTOR; NITISINONE; SODIUM IODIDE SYMPORTER; SUCCINYLACETONE; THYROXINE BINDING GLOBULIN; TYROSINE; CYCLOHEXANONE DERIVATIVE; HYDROLASE; NITROBENZOIC ACID DERIVATIVE;

AMINO ACID METABOLISM; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CELL BASED GENE THERAPY; CELL EXPANSION; CELL ISOLATION; CELL LABELING; CELL LEVEL; CELL PROLIFERATION; CELL TRANSPLANTATION; CONTROLLED STUDY; DISEASE SEVERITY; ENZYME DEFICIENCY; EX VIVO GENE TRANSFER; FEMALE; GENE EXPRESSION; GENETIC TRANSDUCTION; HEREDITARY TYROSINEMIA TYPE 1; IMMUNOHISTOCHEMISTRY; LIVER FIBROSIS; NONHUMAN; NORMAL VALUE; PARTIAL HEPATECTOMY; PIG; POSITRON EMISSION TOMOGRAPHY; PRIORITY JOURNAL; PROMOTER REGION; REPORTER GENE; TYROSINEMIA; ALLOTRANSPLANTATION; ANIMAL; DISEASE MODEL; DRUG EFFECTS; ENZYMOLOGY; GENE THERAPY; GENETICS; LIVER; LIVER CELL; METABOLISM; PROCEDURES;

ANIMALS; CYCLOHEXANONES; DISEASE MODELS, ANIMAL; GENETIC THERAPY; HEPATOCYTES; HYDROLASES; IMMUNOHISTOCHEMISTRY; LIVER; NITROBENZOATES; SWINE; TRANSPLANTATION, HOMOLOGOUS; TYROSINEMIAS;

EID: 84980025204     PISSN: 19466234     EISSN: 19466242     Source Type: Journal    
DOI: 10.1126/scitranslmed.aaf3838     Document Type: Article

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