Life-long correction of hyperbilirubinemia with a neonatal liver-specific AAV-mediated gene transfer in a lethal mouse model of Crigler-Najjar syndrome

Human Gene Therapy

Volumn 25, Issue 9, 2014, Pages 844-855

  Bortolussi, Giulia ^a   Zentillin, Lorena ^a   Vaníkova, Jana ^b   Bockor, Luka ^a   Bellarosa, Cristina ^c   Mancarella, Antonio ^c   Vianello, Eleonora ^c   Tiribelli, Claudio ^c,d   Giacca, Mauro ^a   Vitek, Libor ^b   Muro, Andrés F ^a  

^a INTERNATIONAL CENTRE FOR GENETIC ENGINEERING AND BIOTECHNOLOGY   (Italy)

^b CHARLES UNIVERSITY   (Czech Republic)

^c Centro Studi Fegato   (Italy)

^d UNIVERSITY OF TRIESTE   (Italy)

Author keywords

[No Author keywords available]

Indexed keywords

BILIRUBIN; GLUCURONOSYLTRANSFERASE 1A1; PARVOVIRUS VECTOR; GLUCURONOSYLTRANSFERASE; SERUM ALBUMIN; UGT1A1 ENZYME;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BILIRUBIN BLOOD LEVEL; CLINICAL EFFECTIVENESS; COMPARATIVE STUDY; CONTROLLED STUDY; CRIGLER NAJJAR SYNDROME; EPISOME; GENE; GENE ACTIVITY; GENE EXPRESSION; GENE TRANSFER; HYPERBILIRUBINEMIA; LONG TERM CARE; MOUSE; NEWBORN; NONHUMAN; THERAPY EFFECT; UGT1A1 GENE; ANIMAL; BLOOD; CRIGLER-NAJJAR SYNDROME; DEPENDOPARVOVIRUS; GENE THERAPY; GENE VECTOR; GENETICS; LIVER; METABOLISM; PROCEDURES; REAL TIME POLYMERASE CHAIN REACTION; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION; ROTAROD TEST; SKELETAL MUSCLE; SOUTHERN BLOTTING; WESTERN BLOTTING;

ANIMALS; ANIMALS, NEWBORN; BILIRUBIN; BLOTTING, SOUTHERN; BLOTTING, WESTERN; CRIGLER-NAJJAR SYNDROME; DEPENDOVIRUS; GENE TRANSFER TECHNIQUES; GENETIC THERAPY; GENETIC VECTORS; GLUCURONOSYLTRANSFERASE; LIVER; MICE; MUSCLE, SKELETAL; REAL-TIME POLYMERASE CHAIN REACTION; REVERSE TRANSCRIPTASE POLYMERASE CHAIN REACTION; ROTAROD PERFORMANCE TEST; SERUM ALBUMIN;

EID: 84907285291     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2013.233     Document Type: Article

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