Hematopoietic Stem Cell Gene Therapy: Progress and Lessons Learned

Publication date: 2 November 2017
Source:Cell Stem Cell, Volume 21, Issue 5
Author(s): Richard A. Morgan, David Gray, Anastasia Lomova, Donald B. Kohn
The use of allogeneic hematopoietic stem cells (HSCs) to treat genetic blood cell diseases has become a clinical standard but is limited by the availability of suitable matched donors and potential immunologic complications. Gene therapy using autologous HSCs should avoid these limitations and thus may be safer. Progressive improvements in techniques for genetic correction of HSCs, by either vector gene addition or gene editing, are facilitating successful treatments for an increasing number of diseases. We highlight the progress, successes, and remaining challenges toward the development of HSC gene therapies and discuss lessons they provide for the development of future clinical stem cell therapies.

Teaser

Morgan et al. discuss the progress, successes, and remaining challenges toward the development of hematopoietic stem cell gene therapies and highlight lessons learned and how they can inform the development of future clinical stem cell therapies.


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