Life-Long Correction of Hyperbilirubinemia with a Neonatal Liver-Specific AAV-Mediated Gene Transfer in a Lethal Mouse Model of Crigler

Life-Long Correction of Hyperbilirubinemia with a Neonatal Liver-Specific AAV-Mediated Gene Transfer in a Lethal Mouse Model of Crigler–Najjar Syndrome

Published:2014-09 Issue:9 Volume:25 Page:844-855
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Bortolussi Giulia¹,Zentillin Lorena¹,Vaníkova Jana²,Bockor Luka¹,Bellarosa Cristina³,Mancarella Antonio³,Vianello Eleonora³,Tiribelli Claudio³⁴,Giacca Mauro¹,Vitek Libor²⁵,Muro Andrés F.¹

Affiliation:

1. International Centre for Genetic Engineering and Biotechnology, 34149 Trieste, Italy.

2. Institute of Medical Biochemistry and Laboratory Medicine, First Faculty of Medicine, Charles University, 120 00 Prague, Czech Republic.

3. Centro Studi Fegato, Fondazione Italiana Fegato, AREA Science Park, Campus Basovizza, 34149 Trieste, Italy.

4. Department of Medical Science, University of Trieste, 34128 Trieste, Italy.

5. Fourth Department of Internal Medicine, First Faculty of Medicine, Charles University, 120 00 Prague, Czech Republic.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

http://www.liebertpub.com/doi/pdf/10.1089/hum.2013.233

Reference42 articles.

1. Vascular endothelial growth factor stimulates skeletal muscle regeneration in Vivo

2. Quantitation of transplanted hepatic mass necessary to cure the gunn rat model of hyperbilirubinemia

3. Rescue of bilirubin‐induced neonatal lethality in a mouse model of Crigler‐Najjar syndrome type I by AAV9‐mediated gene transfer

4. Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer

5. Tissue- and Gender-Specific mRNA Expression of UDP-Glucuronosyltransferases (UGTs) in Mice

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