Prolonged liver-specific transgene expression by a non-primate lentiviral vector

Biochemical and Biophysical Research Communications
Reba CondiottiEithan Galun

Abstract

Liver-directed gene therapy has the potential for treatment of numerous inherited diseases affecting metabolic functions. The aim of this study was to evaluate gene expression in hepatocytes using feline immunodeficiency virus-based lentiviral vectors, which may be potentially safer than those based on human immunodeficiency virus. In vitro studies revealed that gene expression was stable for up to 24 days post-transduction and integration into the host cell genome was suggested by Alu PCR and Southern blot analyses. Systemic in vivo administration of viral particles by the hydrodynamics method resulted in high levels of gene expression exclusively in the liver for over 7 months whereas injection of plasmid DNA by the same method led to transient expression levels. Our studies suggest that feline immunodeficiency-based lentiviral vectors specifically transduce liver cells and may be used as a novel vehicle of gene delivery for treatment of metabolic disease.

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Citations

Jan 22, 2009·Molecular Therapy : the Journal of the American Society of Gene Therapy·Kenya KamimuraDexi Liu
Jun 24, 2010·Molecular Therapy : the Journal of the American Society of Gene Therapy·Albert GrinshpunEithan Galun
Jan 10, 2013·Molecular Therapy : the Journal of the American Society of Gene Therapy·Ali NowrouziMike Themis
Aug 29, 2013·Molecular Therapy : the Journal of the American Society of Gene Therapy·Reba CondiottiEithan Galun
Apr 12, 2007·Molecular Therapy : the Journal of the American Society of Gene Therapy·Paula Y P LamKam M Hui
Oct 4, 2007·Molecular Therapy : the Journal of the American Society of Gene Therapy·Takeshi Suda, Dexi Liu
Dec 17, 2008·The AAPS Journal·Kenya Kamimura, Dexi Liu
Oct 7, 2015·Journal of Controlled Release : Official Journal of the Controlled Release Society·Samuel T Crowley, Kevin G Rice
Oct 3, 2008·Biotechnology Journal·Zhijun WangLi Jin
May 26, 2006·The Journal of Gene Medicine·Magdolna G SebestyénJon A Wolff
Nov 27, 2007·Liver International : Official Journal of the International Association for the Study of the Liver·Senthil Kumar VenugopalMark A Zern
Jun 21, 2005·Molecular Therapy : the Journal of the American Society of Gene Therapy·Ela ShaiReba Condiotti
Dec 3, 2008·Cell Transplantation·Elizabeth S FenjvesMichael A Curran
Sep 22, 2006·Gene Therapy·T SudaD Liu
Dec 15, 2006·Gene Therapy·H Herweijer, J A Wolff
Feb 21, 2007·The Journal of Gene Medicine·Clare SeldenHumphrey Hodgson

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