Reprogramming based gene therapy for inherited red blood cell disorders

• 来源期刊: 细胞研究（英文版）
• 年，卷（期）: 2012,（6）
• 页码范围: 941-944
• 页数: 4页
• 语种: 英文
• DOI: 10.1038/cr.2012.54

摘要

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.