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在一项新的研究中,来自美国斯坦福大学医学院的研究人员利用一种被称作CRISPR/Cas9的基因编辑工具修复人干细胞中导致镰状细胞疾病的基因。这是开发一种治疗这种疾病的基因疗法的关键一步。相关研究结果在线发表在Nature期刊上。研究证实这些被修复的干细胞能够制造功能性的血红蛋白分子---在正常的红细胞中,这些分子携带氧气---并且成功地将这些干细胞移植到小鼠体内。他们说这项研究代表着一种修复镰状细胞疾病和地中海贫血等血源性遗传疾病的概念验证。
In a new study, researchers from Stanford University School of Medicine in the US used a gene editing tool called CRISPR / Cas9 to repair genes that cause sickle cell disease in human stem cells. This is a crucial step in developing a gene therapy for the disease. Relevant research results published online in Nature. Studies confirm that these repaired stem cells are capable of producing functional hemoglobin molecules - these molecules carry oxygen in normal red blood cells - and successfully transplanted these stem cells into mice. They say the study represents a proof of concept for repairing blood-borne genetic diseases such as sickle cell disease and thalassemia.