Transcriptional targeting of lentiviral vectors by long terminal repeat enhancer replacement

Journal of Virology
Francesco LottiGiuliana Ferrari

Abstract

Gene therapy of many genetic diseases requires permanent gene transfer into self-renewing stem cells and restriction of transgene expression to specific progenies. Human immunodeficiency virus (HIV)-derived lentiviral vectors are very effective in transducing rare, nondividing stem cell populations (e.g., hematopoietic stem cells) without altering their long-term repopulation and differentiation capacities. We developed a strategy for transcriptional targeting of lentiviral vectors based on replacing the viral long terminal repeat (LTR) enhancer with cell lineage-specific, genomic control elements. An upstream enhancer (HS2) of the erythroid-specific GATA-1 gene was used to replace most of the U3 region of the LTR, immediately upstream of the HIV type 1 (HIV-1) promoter. The modified LTR was used to drive the expression of a reporter gene (the green fluorescent protein [GFP] gene), while a second gene (a truncated form of the p75 nerve growth factor receptor [DeltaLNGFR]) was placed under the control of an internal constitutive promoter to monitor cell transduction, or to immunoselect transduced cells, independently from the expression of the targeted promoter. The transcriptionally targeted vectors were used to transduce cell ...Continue Reading

References

Jul 15, 1992·Proceedings of the National Academy of Sciences of the United States of America·N M SposiS H Orkin
Jul 15, 1995·European Journal of Biochemistry·S H Orkin
Mar 29, 1994·Proceedings of the National Academy of Sciences of the United States of America·P M Challita, D B Kohn
May 14, 1993·Science·R C Mulligan
Aug 1, 1996·Current Opinion in Genetics & Development·D I MartinM Groudine
Nov 12, 1996·Proceedings of the National Academy of Sciences of the United States of America·T ChengD T Scadden
Sep 26, 1997·Nature·I M Verma, N Somia
Nov 5, 1997·Nature Biotechnology·R ZuffereyD Trono
Oct 10, 1998·Journal of Virology·T DullL Naldini
Aug 19, 2000·Molecular Therapy : the Journal of the American Society of Gene Therapy·N KlagesD Trono

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Citations

Jan 24, 2004·Journal of Hepatology·Antonia Follenzi, Sanjeev Gupta
Sep 25, 2008·Molecular Therapy : the Journal of the American Society of Gene Therapy·Francesca Di NunzioFulvio Mavilio
Apr 23, 2009·Molecular Therapy : the Journal of the American Society of Gene Therapy·Fabrizia UrbinatiPunam Malik
Jul 25, 2008·Proceedings of the National Academy of Sciences of the United States of America·Annarita MiccioGiuliana Ferrari
Aug 12, 2003·Human Gene Therapy·Michele De PalmaLuigi Naldini
Jul 28, 2009·Human Gene Therapy·Miguel G ToscanoFrancisco Martín
Jul 5, 2005·Journal of Virology·Susann LuckeKlaus Uberla
Jun 9, 2007·Expert Opinion on Biological Therapy·Sara BobisseAntonio Rosato
Jan 22, 2010·Expert Opinion on Biological Therapy·Sandesh SubramanyaPremlata Shankar
Aug 1, 2015·The Journal of Gene Medicine·Pablo Fernández-RubioIgnacio J Molina
Apr 28, 2009·Advanced Drug Delivery Reviews·Zhihong Dong, Jacques E Nör
Jun 9, 2005·Experimental Eye Research·Sven Christoph BeutelspacherMyra O McClure
Oct 14, 2003·Virology·Edward Reed-Inderbitzin, Wendy Maury
Oct 29, 2004·Molecular Therapy : the Journal of the American Society of Gene Therapy·Loïc DupréMaria-Grazia Roncarolo
Apr 27, 2005·Molecular Therapy : the Journal of the American Society of Gene Therapy·Elisa VignaLuigi Naldini
Jun 6, 2003·Molecular Therapy : the Journal of the American Society of Gene Therapy·Xiaobing YuLinzhao Cheng
May 7, 2003·Gene Therapy·D J Gould, P Favorov
Feb 11, 2010·Neuroscience and Behavioral Physiology·S V Salozhin, A P Bol'shakov
Mar 16, 2021·Molecular Therapy. Methods & Clinical Development·Brenda J SeymourDavid J Rawlings
Nov 3, 2011·Journal of Cell Science·Laura E PerliniFlavia Valtorta

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