In vitro functional correction of hermansky-pudlak syndrome type-1 by lentiviral-mediated gene transfer

Molecular Genetics and Metabolism

Volumn 114, Issue 1, 2015, Pages 62-65

  Ikawa, Yasuhiro ^a   Hess, Richard ^a   Dorward, Heidi ^a   Cullinane, Andrew R ^a   Huizing, Marjan ^a   Gochuico, Bernadette R ^a   Gahl, William A ^a   Candotti, Fabio ^a  

^a NATIONAL HUMAN GENOME RESEARCH INSTITUTE   (United States)

Author keywords

Gene therapy; Hermansky Pudlak syndrome; Lentivirus; Melanocytes; Pulmonary fibrosis

Indexed keywords

PROTEIN; PROTEIN HPS1; UNCLASSIFIED DRUG; HPS1 PROTEIN, HUMAN; HPS4 PROTEIN, HUMAN; MEMBRANE PROTEIN; OXIDOREDUCTASE; TYRP1 PROTEIN, HUMAN;

ARTICLE; CARCINOGENESIS; CONTROLLED STUDY; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER; HUMAN; HUMAN CELL; IN VITRO STUDY; LENTIVIRAL MEDIATED GENE TRANSFER; MELANOCYTE; OCULAR ALBINISM; PROTEIN EXPRESSION; PROTEIN FUNCTION; CELL CULTURE; GENE VECTOR; GENETIC TRANSDUCTION; GENETICS; HERMANSKI-PUDLAK SYNDROME; LENTIVIRINAE; METABOLISM; MUTATION; ULTRASTRUCTURE;

LENTIVIRUS;

CELLS, CULTURED; GENE EXPRESSION; GENETIC THERAPY; GENETIC VECTORS; HERMANSKI-PUDLAK SYNDROME; HUMANS; LENTIVIRUS; MELANOCYTES; MEMBRANE GLYCOPROTEINS; MEMBRANE PROTEINS; MUTATION; OXIDOREDUCTASES; PROTEINS; TRANSDUCTION, GENETIC;

EID: 84920422517     PISSN: 10967192     EISSN: 10967206     Source Type: Journal    
DOI: 10.1016/j.ymgme.2014.11.006     Document Type: Article

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