Journal ArticleDOI
Transduction of Human CD34+ Cells That Mediate Long-Term Engraftment of NOD/SCID Mice by HIV Vectors
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TLDR
A lentiviral vector was able to transduce human CD34+ cells capable of stable, long-term reconstitution of nonobese diabetic/severe combined immunodeficient (NOD/SCID) mice and resulted in transgene expression in multiple lineages of human hematopoietic cells for up to 22 weeks after transplantation.Abstract:
Efficient gene transfer into human hematopoietic stem cells (HSCs) is an important goal in the study of the hematopoietic system as well as for gene therapy of hematopoietic disorders. A lentiviral vector based on the human immunodeficiency virus (HIV) was able to transduce human CD34+ cells capable of stable, long-term reconstitution of nonobese diabetic/severe combined immunodeficient (NOD/SCID) mice. High-efficiency transduction occurred in the absence of cytokine stimulation and resulted in transgene expression in multiple lineages of human hematopoietic cells for up to 22 weeks after transplantation.read more
Citations
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Tat peptide-derivatized magnetic nanoparticles allow in vivo tracking and recovery of progenitor cells.
Maïté Lewin,Nadia Carlesso,Ching-Hsuan Tung,Xiaowu Tang,David G. Cory,David T. Scadden,Ralph Weissleder +6 more
TL;DR: A cell labeling approach using short HIV-Tat peptides to derivatize superparamagnetic nanoparticles is developed, which efficiently internalized into hematopoietic and neural progenitor cells in quantities up to 10–30 pg of super paramagnetic iron per cell.
Journal ArticleDOI
Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy
Nathalie Cartier,Salima Hacein-Bey-Abina,Cynthia C. Bartholomae,Gabor Istvan Veres,Manfred Schmidt,Ina Kutschera,Michel Vidaud,Ulrich Abel,Liliane Dal-Cortivo,Laure Caccavelli,Nizar Mahlaoui,Veronique Kiermer,Denice Mittelstaedt,Céline Bellesme,Najiba Lahlou,François Lefrère,Stéphane Blanche,Muriel Audit,Emmanuel Payen,Philippe Leboulch,Philippe Leboulch,Bruno l’Homme,Pierre Bougnères,Christof von Kalle,Alain Fischer,Marina Cavazzana-Calvo,Patrick Aubourg +26 more
TL;DR: Lentiviral-mediated gene therapy of hematopoietic stem cells can provide clinical benefits in ALD, and progressive cerebral demyelination in the two patients stopped, a clinical outcome comparable to that achieved by allogeneic HCT.
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Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics
TL;DR: Further vector refinement and/or development is required before gene therapy will become standard care for any individual disorder, and some clinical successes are over the horizon.
Journal ArticleDOI
Humanized mice in translational biomedical research
TL;DR: This Review discusses the development of these new generations of humanized mice, how they will facilitate translational research in several biomedical disciplines and approaches to overcome the remaining limitations of these models.
Journal ArticleDOI
Gene transfer by lentiviral vectors is limited by nuclear translocation and rescued by HIV-1 pol sequences.
TL;DR: The results indicate that nuclear translocation of the genome is a rate-limiting step in lentiviral infection of both dividing and non-dividing cells, and that it depends on protein and nucleic acid sequence determinants.
References
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Journal ArticleDOI
In Vivo Gene Delivery and Stable Transduction of Nondividing Cells by a Lentiviral Vector
Luigi Naldini,Ulrike Blömer,Philippe Gallay,Daniel S. Ory,Richard C. Mulligan,Fred H. Gage,Inder M. Verma,Didier Trono +7 more
TL;DR: The ability of HIV-based viral vectors to deliver genes in vivo into nondividing cells could increase the applicability of retroviral vectors in human gene therapy.
Journal ArticleDOI
Gene therapy -- promises, problems and prospects
Inder M. Verma,Nikunj V. Somia +1 more
TL;DR: The prospects are good — by the year 2010, gene therapy may be as routine a practice as heart transplants are today.
Journal ArticleDOI
Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo.
TL;DR: An HIV vector system in which the virulence genes env, vif, vpr, vpu, and nef have been deleted is described, and this multiply attenuated vector conserved the ability to transduce growth-arrested cells and monocyte-derived macrophages in culture, and could efficiently deliver genes in vivo into adult neurons.
Journal ArticleDOI
Development of a Self-Inactivating Lentivirus Vector
TL;DR: Injection of viruses into the rat brain showed that a SIN vector containing the green fluorescent protein gene under the control of the internal CMV promoter transduced neurons as efficiently as a wild-type vector, indicating that the HIV-1 LTR promoter is transcriptionally active in neurons even in the absence of Tat.
Journal Article
Multiple defects in innate and adaptive immunologic function in NOD/LtSz-scid mice.
Leonard D. Shultz,Peter A. Schweitzer,Sherri W. Christianson,Bruce Gott,Isabelle B. Schweitzer,B Tennent,S D McKenna,L Mobraaten,Thiruchandurai V. Rajan,Dale L. Greiner +9 more
TL;DR: The multiple defects in innate and adaptive immunity unique to the NOD/LtSz-scid/scid mouse provide an excellent in vivo environment for reconstitution with human hematopoietic cells.