Reprogramming based gene therapy for inherited red blood cell disorders

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 genet...

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Veröffentlicht in:	Cell research 2012-06, Vol.22 (6), p.941-944
Hauptverfasser:	Xu, Xiuling, Qu, Jing, Suzuki, Keiichiro, Li, Mo, Zhang, Weizhou, Liu, Guang-Hui, Belmonte, Juan Carlos Izpisua
Format:	Artikel
Sprache:	eng
Schlagworte:	631/532/2064/2158 692/699/1541 692/700/565/2319 Anemia, Sickle Cell - genetics Anemia, Sickle Cell - pathology beta-Globins - genetics beta-Globins - metabolism Biomedical and Life Sciences Cell Biology Cellular Reprogramming Genetic Therapy Hematologic Diseases - genetics Hematologic Diseases - therapy Hematopoietic Stem Cell Transplantation Humans Induced Pluripotent Stem Cells - cytology Induced Pluripotent Stem Cells - metabolism Life Sciences Research Highlight Thalassemia - genetics Thalassemia - metabolism Thalassemia - pathology 基因治疗多能干细胞红细胞红血细胞造血干细胞移植造血系统遗传性疾病重新编程
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Zusammenfassung:	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. Red blood cells （RBCs） are unique, highly specialized, and the most abundant cells in humans and other organisms. The primary function of RBCs is transportation of the respiratory gases O, and CO2. Meanwhile,
ISSN:	1001-0602 1748-7838
DOI:	10.1038/cr.2012.54