Hematopoietic stem cell engineering at a crossroads Journal Article


Authors: Riviere, I.; Dunbar, C. E.; Sadelain, M.
Article Title: Hematopoietic stem cell engineering at a crossroads
Abstract: The genetic engineering of hematopoietic stem cells is the basis for potentially treating a large array of hereditary and acquired diseases, and stands as the paradigm for stem cell engineering in general. Recent clinical reports support the formidable promise of this approach but also highlight the limitations of the technologies used to date, which have on occasion resulted in clonal expansion, myelodysplasia, or leukemogenesis. New research directions, predicated on improved vector designs, targeted gene delivery or the therapeutic use of pluripotent stem cells, herald the advent of safer and more effective hematopoietic stem cell therapies that may transform medical practice. In this review, we place these recent advances in perspective, emphasizing the solutions emerging from a wave of new technologies and highlighting the challenges that lie ahead.
Keywords: review; myelodysplastic syndrome; genetic engineering; clonal variation; leukemogenesis; safety; pluripotent stem cell; hematopoietic stem cell; technology; gene delivery system; cell engineering
Journal Title: Blood
Volume: 119
Issue: 5
ISSN: 0006-4971
Publisher: American Society of Hematology  
Date Published: 2012-02-02
Start Page: 1107
End Page: 1116
Language: English
DOI: 10.1182/blood-2011-09-349993
PROVIDER: scopus
PUBMED: 22096239
PMCID: PMC3277348
DOI/URL:
Notes: --- - "Export Date: 1 March 2012" - "CODEN: BLOOA" - "Source: Scopus"
Altmetric
Citation Impact
BMJ Impact Analytics
MSK Authors
  1. Michel W J Sadelain
    583 Sadelain
  2. Isabelle C Riviere
    240 Riviere