Research work · 2017
Hematopoietic Stem Cell Gene Therapy –Progress and Lessons Learned
Cell stem cell
Abstract
The use of allogeneic hematopoietic stem cells (HSCs) to treat genetic blood cell diseases has become a clinical standard but is limited by 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 development of HSC gene therapies and discuss lessons they provide for development of future clinical stem cell therapies.