Issues in the manufacture and transplantation of genetically modified hematopoietic stem cells Journal Article


Authors: Sadelain, M.; Frassoni, F.; Rivière, I.
Article Title: Issues in the manufacture and transplantation of genetically modified hematopoietic stem cells
Abstract: The advent of safe and practical means to correct, enhance or protect blood cells at the genetic level offers tantalizing therapeutic perspectives. At present, gene delivery using a replication-defective retrovirus is the most efficient method to stably transduce hematopoietic cells. The successful adaptation of retroviral infection to hematopoietic stem cells requires optimized transduction conditions that maximize gene transfer while preserving the cells' potential for engraftment and long-term hematopoiesis. The successful establishment of effective transduction protocols hinges on retrovirus biology as well as stem cell and transplantation biology. Interestingly, the genetic approach could permit novel strategies to promote host repopulation by transplanted stem cells. However, regulated and predictable expression of any transgene integrated at random chromosomal locations cannot be taken for granted. Investigation of the control of transgene expression and prevention of vector silencing will become increasingly important. (C) 2000 Lippincott Williams and Wilkins, Inc.
Keywords: review; animals; gene targeting; gene expression; hematopoietic stem cell transplantation; cell population; gene transfer; genetic transduction; transduction, genetic; dna modification; genetic recombination; genetic engineering; transgene; hematopoietic stem cells; hematopoiesis; virus infection; hematopoietic stem cell; retrovirus; retroviridae; virus vector; humans; priority journal
Journal Title: Current opinion in Hematology
Volume: 7
Issue: 6
ISSN: 1065-6251
Publisher: Lippincott Williams & Wilkins, Ltd.  
Date Published: 2000-11-01
Start Page: 364
End Page: 377
Language: English
DOI: 10.1097/00062752-200011000-00008
PUBMED: 11055510
PROVIDER: scopus
DOI/URL:
Notes: Export Date: 18 November 2015 -- Source: Scopus
Altmetric Score
MSK Authors
  1. Michel W J Sadelain
    462 Sadelain
  2. Isabelle C Riviere
    168 Riviere