Evaluation of engineered AAV capsids for hepatic factor IX gene transfer in murine and canine models

Journal of Translational Medicine

Volumn 15, Issue 1, 2017, Pages

  Markusic, David M ^a   Nichols, Timothy C ^b   Merricks, Elizabeth P ^b   Palaschak, Brett ^a   Zolotukhin, Irene ^a   Marsic, Damien ^a   Zolotukhin, Sergei ^a   Srivastava, Arun ^a   Herzog, Roland W ^a  

^a UNIVERSITY OF FLORIDA   (United States)

^b UNIVERSITY OF NORTH CAROLINA SCHOOL OF MEDICINE   (United States)

Author keywords

AAV; Capsid; Factor IX; Gene therapy; Hemophilia B

Indexed keywords

ADENO ASSOCIATED VIRUS VECTOR; BLOOD CLOTTING FACTOR 9; LYSINE; TYROSINE;

ADENO ASSOCIATED VIRUS 2; AMINO ACID SUBSTITUTION; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CANINE MODEL; CONTROLLED STUDY; GENE EXPRESSION; GENE MUTATION; HEMOPHILIA B; IN VIVO GENE TRANSFER; LIVER; MALE; MOUSE; MURINE MODEL; NONHUMAN; VIRAL GENE THERAPY; VIRUS CAPSID; ANIMAL; BAGG ALBINO MOUSE; C57BL MOUSE; DEPENDOPARVOVIRUS; DOG; GENE TRANSFER; GENE VECTOR; GENETIC ENGINEERING; GENETIC TRANSDUCTION; GENETICS; LIVER CELL; METABOLISM; MUTATION;

ANIMALS; CAPSID; DEPENDOVIRUS; DOGS; FACTOR IX; GENE TRANSFER TECHNIQUES; GENETIC ENGINEERING; GENETIC VECTORS; HEMOPHILIA B; HEPATOCYTES; LIVER; LYSINE; MALE; MICE, INBRED BALB C; MICE, INBRED C57BL; MODELS, ANIMAL; MUTATION; TRANSDUCTION, GENETIC; TYROSINE;

EID: 85018440394     PISSN: None     EISSN: 14795876     Source Type: Journal    
DOI: 10.1186/s12967-017-1200-1     Document Type: Article

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