CTX001输注几乎消除了所有TDT患者的输血,并消除了所有SCD患者的VOC,相关的有临床意义的HbF和总Hb增加随时间维持。CRISPR/Cas9编辑的BCL11A等位基因的比例在>1年后保持稳定,表明长期HSC被成功编辑,为观察到的显著、持久的疗效提供了基础。安...
在那里，他们用CRISPR进行编辑，精确切割一个名为BCL11A的基因位点，然后干细胞被运回治疗医院。
Then, CRISPR was used to edit the BCL11A gene, repressor of fetal hemoglobin (Hbf) expression, in their cells. Restoring fetal hemoglobin levels could circumvent the harmful effects of deleterious mutations in both diseases and afford a normal life for patients. These edited stem cells were ...
It uses CRISPR/Cas9 to make a patient’s haematopoietic stem cells produce high levels of foetal haemoglobin (HbF) in red blood cells, by introducing a gene known as BCL11A which down-regulates the production of the adult form of the oxygen-carrying molecule. Reverting to HbF in thalassaemia...