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Reprogramming based gene therapy for inherited red blood cell disorders
Journal article   Open access   Peer reviewed

Reprogramming based gene therapy for inherited red blood cell disorders

Xiuling Xu, Jing Qu, Keiichiro Suzuki, Mo Li, Weizhou Zhang, Guang-Hui Liu and Juan Carlos Izpisua Belmonte
Cell research, Vol.22(6), pp.941-944
06/2012
DOI: 10.1038/cr.2012.54
PMCID: PMC3367524
PMID: 22473006
url
https://doi.org/10.1038/cr.2012.54View
Published (Version of record) Open Access

Abstract

Hematopoietic stem cell transplantation remains the only curative therapy for certain genetic diseases of the hematopoietic system, including some inheritable deficiencies in red blood cells (RBC). Recent advances in induced pluripotent stem cells may open a new era for the cure of such severe genetic RBC diseases.
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