碱基编辑增加镰状细胞病中的血红蛋白

IF 33.1 1区 生物学 Q1 BIOTECHNOLOGY & APPLIED MICROBIOLOGY
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引用次数: 0

摘要

Beam Therapeutics公司关于其碱基编辑疗法Beam -101的早期临床数据首次显示,该技术可以恢复镰状细胞病患者的功能性血红蛋白。在接受BEAM-101治疗的4例患者中,在随访1-6个月时,功能性胎儿血红蛋白水平增加到60%以上,患者红细胞镰状细胞减少,细胞粘连减少,血流特性改善。此外,没有患者有镰状细胞病典型的痛苦的血管闭塞危象。Beam将在12月的美国血液学学会会议上公布进一步的数据。BEAM-101利用腺嘌呤碱基编辑器在离体患者干细胞中引入γ-珠蛋白基因HBG1和HBG2启动子区域的单碱基变化。这些变化破坏了BCL11A抑制因子的结合,增加了胎儿血红蛋白的表达。如果获得批准,BEAM-101将与Vertex的crispr疗法Casgevy和bluebird bio的基因疗法Lyfgenia (lovotibeglogene autotemcell)一起使用。在单独的试验中,这些镰状细胞疗法在两年后将功能性血红蛋白提高到40%左右。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Base editing boosts hemoglobin in sickle cell disease
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来源期刊
Nature biotechnology
Nature biotechnology 工程技术-生物工程与应用微生物
CiteScore
63.00
自引率
1.70%
发文量
382
审稿时长
3 months
期刊介绍: Nature Biotechnology is a monthly journal that focuses on the science and business of biotechnology. It covers a wide range of topics including technology/methodology advancements in the biological, biomedical, agricultural, and environmental sciences. The journal also explores the commercial, political, ethical, legal, and societal aspects of this research. The journal serves researchers by providing peer-reviewed research papers in the field of biotechnology. It also serves the business community by delivering news about research developments. This approach ensures that both the scientific and business communities are well-informed and able to stay up-to-date on the latest advancements and opportunities in the field. Some key areas of interest in which the journal actively seeks research papers include molecular engineering of nucleic acids and proteins, molecular therapy, large-scale biology, computational biology, regenerative medicine, imaging technology, analytical biotechnology, applied immunology, food and agricultural biotechnology, and environmental biotechnology. In summary, Nature Biotechnology is a comprehensive journal that covers both the scientific and business aspects of biotechnology. It strives to provide researchers with valuable research papers and news while also delivering important scientific advancements to the business community.
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