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Hematopoietic stem cell engineering at a crossroads

Rivière, Isabelle, Dunbar, Cynthia E., Sadelain, Michel·2012·Blood
DOI 10.1182/blood-2011-09-349993
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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.

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Cited by

See all 54

Metadata

Authors
Rivière, I., Dunbar, C. E., & Sadelain, M.
Affiliations
  • Center for Cell Engineering, Molecular Pharmacology and Chemistry Program, Memorial Sloan-Kettering Cancer Center, New York, NY; and
  • Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD
Venue
Blood
Publisher
American Society of Hematology
Published
2012-02-02
Volume
119
Issue
5
Pages
1107-1116
ISSN
  • 0006-4971
  • 1528-0020
DOI
10.1182/blood-2011-09-349993
Cited by
54
References
18
Language
en

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