Treatment of phenylketonuria using minicircle-based naked-DNA gene transfer to murine liver-Reference-Cited by-同舟云学术

Treatment of phenylketonuria using minicircle-based naked-DNA gene transfer to murine liver

Published:2014-07-29 Issue:3 Volume:60 Page:1035-1043
ISSN:0270-9139
Container-title:Hepatology
language:en
Short-container-title:Hepatology

Author:

Viecelli Hiu Man¹,Harbottle Richard P.²,Wong Suet Ping²,Schlegel Andrea³,Chuah Marinee K.⁴⁵,VandenDriessche Thierry⁴⁵,Harding Cary O.⁶,Thöny Beat¹

Affiliation:

1. Division of Metabolism, Department of Pediatrics; University of Zurich, Zurich, Switzerland; and affiliated with the Children's Research Center Zurich

2. Section of Molecular Medicine, National Heart and Lung Institute; Imperial College London; London United Kingdom

3. Swiss HPB and Transplant Center, Department of Surgery; University Hospital Zurich; Zurich Switzerland

4. Department of Gene Therapy & Regenerative Medicine; Free University of Brussels; Brussels Belgium

5. Department of Cardiovascular Sciences, Center for Molecular and Vascular Biology; University of Leuven; Leuven Belgium

6. Departments of Molecular and Medical Genetics and Pediatrics; Oregon Health & Science University; Portland OR USA

Funder

Swiss National Science Foundation

National Institutes of Health

Publisher

Wiley

Subject

Hepatology

Link

http://onlinelibrary.wiley.com/wol1/doi/10.1002/hep.27104/fullpdf

Reference47 articles.

1. Phenylketonuria;Blau;Lancet,2010

2. Mouse models of human phenylketonuria;Shedlovsky;Genetics,1993

3. Administration-route and gender-independent long-term therapeutic correction of phenylketonuria (PKU) in a mouse model by recombinant adeno-associated virus 8 pseudotyped vector-mediated gene transfer;Ding;Gene Ther,2006

4. Complete restoration of phenylalanine oxidation in phenylketonuria mouse by a self-complementary adeno-associated virus vector;Yagi;J Gene Med,2011

5. Correction of murine PKU following AAV-mediated intramuscular expression of a complete phenylalanine hydroxylating system;Ding;Mol Ther,2008

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