A top-down approach for construction of hybrid polymer-virus gene delivery vectors

Journal of Controlled Release : Official Journal of the Controlled Release Society
Joshua D RamseyDaniel W Pack

Abstract

Safe and efficient delivery of therapeutic nucleic acids remains the primary hurdle for human gene therapy. While many researchers have attempted to re-engineer viruses to be suited for gene delivery, others have sought to develop non-viral alternatives. We have developed a complementary approach in which viral and synthetic components are combined to form hybrid nanoparticulate vectors. In particular, we complexed non-infectious retrovirus-like particles lacking a viral envelope protein, from Moloney murine leukemia virus (M-VLP) or human immunodeficiency virus (H-VLP), with poly-L-lysine (PLL) or polyethylenimine (PEI) over a range of polymer/VLP ratios. At appropriate stoichiometry (75-250 microg polymer/10(6) VLP), the polymers replace the function of the viral envelope protein and interact with the target cell membrane, initiate cellular uptake and facilitate escape from endocytic vesicles. The viral particle, once in the cytosol, efficiently completes its normal infection process including integration of viral genes with the host genome as demonstrated by long-term (at least 5 weeks) transgene expression. In addition, hybrid vectors comprising H-VLP were shown to be capable of infecting non-dividing cells.

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Citations

Jun 2, 2011·Molecular Therapy : the Journal of the American Society of Gene Therapy·Jae-Hyung JangLonnie D Shea
Oct 26, 2013·Nanomedicine·Seog-Jin SeoHae-Won Kim
Jul 11, 2014·Journal of Controlled Release : Official Journal of the Controlled Release Society·Rahul KeswaniDaniel W Pack
Mar 15, 2013·Molecular Pharmaceutics·Rahul K KeswaniDaniel W Pack
Jan 16, 2016·Advanced Healthcare Materials·Matteo PalmaManus J Biggs
Dec 21, 2010·Journal of Controlled Release : Official Journal of the Controlled Release Society·Soo Kyung Cho, Young Jik Kwon
May 29, 2013·Journal of Biomedical Materials Research. Part a·Kumar SingarapuJoshua D Ramsey
Apr 23, 2013·Journal of Virological Methods·Sravanthi VupputuriJoshua D Ramsey
Apr 18, 2015·Journal of Controlled Release : Official Journal of the Controlled Release Society·Rahul K KeswaniDaniel W Pack
Sep 3, 2013·Journal of Controlled Release : Official Journal of the Controlled Release Society·Jacob J ElmerKaushal Rege
Aug 9, 2013·Molecular Pharmaceutics·Jing XuJoseph M DeSimone
Feb 10, 2018·The Journal of Gene Medicine·Pratheppa RajagopalUma Maheswari Krishnan
Jan 1, 2014·Journal of Materials Chemistry. B, Materials for Biology and Medicine·Charles H JonesBlaine A Pfeifer
Dec 4, 2019·Wiley Interdisciplinary Reviews. Nanomedicine and Nanobiotechnology·Janelle W SalamehMarxa L Figueiredo
Jul 3, 2018·Oncology Letters·Tinglu LiHe Huang
Oct 8, 2020·Biomaterials Science·Kübra Kaygisiz, Christopher V Synatschke

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