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

Michel Sadelain, Francesco Frassoni, Isabelle Rivière

Research output: Contribution to journalArticlepeer-review


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.

Original languageEnglish
Pages (from-to)364-377
Number of pages14
JournalCurrent Opinion in Hematology
Issue number6
Publication statusPublished - 2000

ASJC Scopus subject areas

  • Hematology


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