PMID: 7538667May 9, 1995Paper

Long-term in vivo expression of the human glucocerebrosidase gene in nonhuman primates after CD34+ hematopoietic cell transduction with cell-free retroviral vector preparations

Proceedings of the National Academy of Sciences of the United States of America
L C XuR O Brady

Abstract

Successful gene transfer into stem cells would provide a potentially useful therapeutic modality for treatment of inherited and acquired disorders affecting hematopoietic tissues. Coculture of primate bone marrow cells with retroviral producer cells, autologous stroma, or an engineered stromal cell line expressing human stem cell factor has resulted in a low efficiency of gene transfer as reflected by the presence of 0.1-5% of genetically modified cells in the blood of reconstituted animals. Our experiments in a nonhuman primate model were designed to explore various transduction protocols that did not involve coculture in an effort to define clinically useful conditions and to enhance transduction efficiency of repopulating cells. We report the presence of genetically modified cells at levels ranging from 0.1% (granulocytes) to 14% (B lymphocytes) more than 1 year following reconstitution of myeloablated animals with CD34+ immunoselected cells transduced in suspension culture with cytokines for 4 days with a retrovirus containing the glucocerebrosidase gene. A period of prestimulation for 7 days in the presence of autologous stroma separated from the CD34+ cells by a porous membrane did not appear to enhance transduction effic...Continue Reading

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Citations

Sep 11, 2002·The Journal of Gene Medicine·Bruce E Torbett
Mar 1, 1996·Lipids·R O Brady, N W Barton
May 7, 1997·Transfusion Science·B Péault, P Charbord
Dec 19, 1995·Proceedings of the National Academy of Sciences of the United States of America·M MigitaS Karlsson
Nov 13, 2002·Journal of Hematotherapy & Stem Cell Research·Amrita KrishnanArturo Molina
Sep 10, 1996·Human Gene Therapy·V W Van Beusechem, D Valerio
Oct 10, 1997·Human Gene Therapy·J DybingP Anklesaria
Jun 1, 1996·Journal of Medical Primatology·M RosenzweigR P Johnson
Jan 1, 1997·Stem Cells·M HavengaH H van Es
Nov 4, 2000·Expert Opinion on Investigational Drugs·H A James
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Oct 8, 2003·Molecular Therapy : the Journal of the American Society of Gene Therapy·Kaoru YamadaChristopher E Walsh
Jun 8, 2006·The Journal of Veterinary Medical Science·Naohide AgeyamaKeiji Terao
Aug 10, 2000·Molecular Therapy : the Journal of the American Society of Gene Therapy·D A HeimC E Dunbar

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