基于编辑的CAR7 T细胞用于复发的T细胞急性淋巴细胞白血病
Robert Chiesa1, Christos Georgiadis1, Farhatullah Syed1
1From Great Ormond Street Hospital for Children NHS Trust (R.C., G.O., T.B., J.C., S.A., R.T., K.G., D.O., A.V., W.Q.) and the UCL Great Ormond Street Institute of Child Health (C.G., F.S., H.Z., A.E., S.A.G., R.P., A.K., W.Q.) - both in London.
The New England journal of medicine
|June 14, 2023
概括
基因编辑精确地转换DNA核酸,使基因无休息地无活化. 这种基于CRISPR的方法产生了有效的化学抗原受体T细胞,用于治疗儿童复发性白血病.
科学领域:
- 以CRISPR为媒介的细胞因子去胺,用于精确的DNA基编辑.
- 开发通用,现成的仿真抗原受体 (CAR) T 细胞.
背景情况:
- 通过CRISPR进行的cytidine deamination可以实现有针对性的C-to-T转换,而不会破坏DNA.
- 这种精确的基因编辑避免了染色体异常,使其适合治疗应用.
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