Progress and Perspectives in the Development of Lentiviral Vector Producer Cells.

Biotechnology Journal
Mariana V FerreiraAna S Coroadinha

Abstract

After two decades of clinical trials, gene therapy demonstrated effectiveness in the treatment of a series of diseases. Currently, several gene therapy products are approved and used in the clinic. Lentiviral vectors (LVs) are one of the most used transfer vehicles to deliver genetic material and the vector of choice to modify hematopoietic cells to correct primary immunodeficiencies, hemoglobinopathies, and leukodystrophies. LVs are also widely used to modify T cells to treat cancers in immunotherapies (e.g., chimeric antigen receptors T cell therapies, CAR-T). In genome editing, LVs are used to deliver sequence-specific designer nucleases and DNA templates. The approval LV gene therapy products (e.g., Kymriah, for B-cell Acute lymphoblastic leukemia treatment; LentiGlobin, for β-thalassemia treatment) reinforced the need to improve their bioprocess manufacturing. The production has been mostly dependent on transient transfection. Production from stable cell lines facilitate GMP compliant processes, providing an easier scale-up, reproducibility and cost-effectiveness. The establishment of stable LV producer cell lines presents, however, several difficulties, with the cytotoxicity of some of the vector proteins being a major ch...Continue Reading

References

Jun 15, 1992·Proceedings of the National Academy of Sciences of the United States of America·M Gossen, H Bujard
Apr 1, 1973·Virology·F L Graham, A J van der Eb
Aug 1, 1995·Proceedings of the National Academy of Sciences of the United States of America·O BoussifJ P Behr
Apr 16, 1996·Proceedings of the National Academy of Sciences of the United States of America·D NoR M Evans
Jul 1, 1996·Journal of Virology·H YuJ P Dougherty
Oct 10, 1998·Journal of Virology·T DullL Naldini
Dec 16, 1998·Journal of Virology·T KafriI M Verma
Feb 17, 2000·Trends in Biotechnology·A Mountain
Feb 13, 2001·Molecular Therapy : the Journal of the American Society of Gene Therapy·K XuT Kafri
Apr 26, 2001·Molecular Therapy : the Journal of the American Society of Gene Therapy·S SparacioV Bosch
Jul 12, 2002·Current Gene Therapy·L J Chang, E E Gay
Apr 8, 2003·Nature Biotechnology·Yasuhiro IkedaMary Collins
Oct 21, 2004·Molecular Cell·Melanie GallaChristopher Baum
Nov 16, 2004·Journal of Virological Methods·Miguel Sena-EstevesAlan W Flake
Aug 17, 2005·Current Drug Targets. CNS and Neurological Disorders·Maria C Pedroso de LimaLuís Pereira de Almeida
Mar 7, 2006·Molecular Therapy : the Journal of the American Society of Gene Therapy·Adam S CockrellTal Kafri
Mar 25, 2006·Molecular Therapy : the Journal of the American Society of Gene Therapy·Sarah J NightingaleDonald B Kohn
Nov 7, 2006·BMC Biotechnology·Alaka MullickBernard Massie
Feb 20, 2007·Current Gene Therapy·Frédéric Pâques, Philippe Duchateau
Jan 9, 2008·Molecular Therapy : the Journal of the American Society of Gene Therapy·Sophie BroussauBernard Massie
Oct 17, 2009·The AAPS Journal·Mohammed S Al-Dosari, Xiang Gao
Feb 2, 2010·Journal of Controlled Release : Official Journal of the Controlled Release Society·Tao GengChang Lu
Nov 9, 2010·Current Gene Therapy·M Schweizer, O-W Merten
Apr 18, 2012·The Biochemical Journal·Toshie SakumaYasuhiro Ikeda
Jun 19, 2013·Human Gene Therapy Methods·Anna StornaiuoloChiara Bovolenta
Jul 17, 2013·Molecular Therapy : the Journal of the American Society of Gene Therapy·Alok V JoglekarDonald B Kohn
Aug 3, 2013·Nucleic Acids Research·Luhan YangGeorge Church
Mar 7, 2014·The New England Journal of Medicine·Pablo TebasCarl H June

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