Modular protein engineering for non-viral gene therapy

Trends in Biotechnology
A Arís, A Villaverde

Abstract

Despite the recognized potential of viral vectors for gene therapy, growing biological concerns are prompting the exploration of safer, non-viral vectors to deliver therapeutic nucleic acids. In this context, recombinant proteins can be bioproduced on a large scale, without the need for further in vitro modifications, being free of known or suspected biohazards. For these vehicles to act as efficient gene-delivery devices, they must perform relevant functions that mimic those of viruses; namely, nucleic acid condensation, targeted cell attachment and internalization, endosomal escape and nuclear transfer. Modular engineering enables the construction of chimeric polypeptides in which selected domains, potentially from different origins, provide the required activities. An equilibrate combination and spatial distribution of such partner elements has generated promising prototypes, able to deliver expressible DNA to tissue culture but also to specific cell-types in whole organisms.

References

Apr 1, 1992·Experimental Cell Research·A A RosenkranzA S Sobolev
Jul 1, 1992·Proceedings of the National Academy of Sciences of the United States of America·E WagnerM L Birnstiel
May 15, 1991·Proceedings of the National Academy of Sciences of the United States of America·E WagnerM L Birnstiel
Apr 26, 1994·Proceedings of the National Academy of Sciences of the United States of America·J C PeralesR W Hanson
Jan 9, 1996·Proceedings of the National Academy of Sciences of the United States of America·T FerkolR W Hanson
Aug 1, 1996·Human Gene Therapy·J D FritzJ A Wolff
Jun 1, 1997·Nature Biotechnology·R PasqualiniE Ruoslahti
Jul 1, 1997·Human Gene Therapy·R W PaulR W Overell
Jan 27, 1998·The Journal of Biological Chemistry·A EfthymiadisD A Jans
Apr 29, 1998·The Journal of Biological Chemistry·H PollardD Escande
Jun 6, 1998·Trends in Microbiology·D J Evans, J W Almond
Sep 17, 1998·FEBS Letters·J X Feliu, A Villaverde
Oct 17, 1998·The Journal of Biological Chemistry·D V Schaffer, D A Lauffenburger
Nov 2, 1999·Biochemical and Biophysical Research Communications·P D KassnerD Larocca
Dec 22, 1999·Protein Expression and Purification·R S PasquinelliM M Ataai
Jan 29, 2000·Biotechnology and Bioengineering·D V SchafferD A Lauffenburger
Feb 7, 2001·Current Opinion in Biotechnology·N Wu, M M Ataai
Sep 23, 2000·Gene Therapy·E FortunatiB J Scholte
Oct 12, 2000·FASEB Journal : Official Publication of the Federation of American Societies for Experimental Biology·Y SatoY Niitsu
Nov 10, 2000·Advanced Drug Delivery Reviews·C Uherek, W Wels
Nov 22, 2000·Brain Research. Brain Research Protocols·D Martinez-Fong, I Navarro-Quiroga
Dec 1, 2000·Biochemical and Biophysical Research Communications·A Arís, A Villaverde
Feb 24, 2001·The Journal of Gene Medicine·J FominayaA Bernad
Nov 16, 2001·Molecular Therapy : the Journal of the American Society of Gene Therapy·R C CarlisleL W Seymour
Oct 10, 2002·Biochemical and Biophysical Research Communications·Reetika GaurYogendra Singh
Oct 18, 2002·Biopolymers·Olulanu H AinaKit S Lam

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Citations

Aug 25, 2007·Nanomedicine·Raymond M SchiffelersGert Storm
Apr 6, 2006·Microbial Cell Factories·Rosa María FerrazAntonio Villaverde
Jan 5, 2011·Microbial Cell Factories·Esther Vázquez, Antonio Villaverde
Aug 3, 2011·Microbial Cell Factories·Esther VazquezAntonio Villaverde
Apr 1, 2005·Drug Discovery Today. Technologies·Enrico MastrobattistaDaan J A Crommelin
Nov 26, 2013·Nanomedicine : Nanotechnology, Biology, and Medicine·Ugutz UnzuetaEsther Vázquez
Jan 31, 2012·Journal of Controlled Release : Official Journal of the Controlled Release Society·Marcelo A S ToledoAdriano R Azzoni
Apr 4, 2009·Biotechnology Advances·Paolo SaccardoNuria González-Montalbán
Mar 25, 2008·Trends in Biotechnology·Neus Ferrer-MirallesAntonio Villaverde
Jun 4, 2014·Journal of Peptide Science : an Official Publication of the European Peptide Society·Stefania GaldieroMassimiliano Galdiero
Jul 16, 2014·International Journal of Pharmaceutics·Mireia PesarrodonaEsther Vazquez
Oct 10, 2013·Critical Reviews in Biotechnology·Neus Ferrer-MirallesAntonio Villaverde
Mar 26, 2015·Trends in Biotechnology·Ugutz UnzuetaAntonio Villaverde
Nov 13, 2004·Drug Discovery Today·Martin R Stocks
May 26, 2016·Scientific Reports·Wenlong BaoYinglang Wan
Mar 9, 2006·Nature Reviews. Drug Discovery·Enrico MastrobattistaDaan J A Crommelin
Aug 28, 2007·Annals of Neurology·Hugo PeluffoBerta González
Dec 13, 2005·Journal of Fluorescence·Mamata H Kombrabail, G Krishnamoorthy
May 28, 2005·Journal of Biomedical Materials Research. Part a·Huihua FuJeffrey O Hollinger
Oct 13, 2009·Biomacromolecules·Matthew L DaviesPeter Douglas
Mar 2, 2005·Biochemical and Biophysical Research Communications·Andrea VeraAntonio Villaverde

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