Treating sickle cell anemia.
作者信息
Tisdale John F, Thein Swee Lay, Eaton William A
机构信息
Cellular and Molecular Therapeutics Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD 20892, USA.
Sickle Cell Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD 20892, USA.
出版信息
Science. 2020 Mar 13;367(6483):1198-1199. doi: 10.1126/science.aba3827.
Abstract
摘要
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Treating sickle cell anemia.治疗镰状细胞贫血。
Science. 2020 Mar 13;367(6483):1198-1199. doi: 10.1126/science.aba3827.
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Effect of donor type and conditioning regimen intensity on allogeneic transplantation outcomes in patients with sickle cell disease: a retrospective multicentre, cohort study.供体类型和预处理方案强度对镰状细胞病患者异基因移植结局的影响：一项回顾性多中心队列研究
Lancet Haematol. 2019 Nov;6(11):e585-e596. doi: 10.1016/S2352-3026(19)30154-1. Epub 2019 Sep 5.
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Lentiviral and genome-editing strategies for the treatment of β-hemoglobinopathies.慢病毒和基因组编辑策略治疗β-血红蛋白病。
Blood. 2019 Oct 10;134(15):1203-1213. doi: 10.1182/blood.2019000949.
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A Phase 3 Randomized Trial of Voxelotor in Sickle Cell Disease.一项针对镰状细胞病患者的 voxotor 的 3 期随机试验。
N Engl J Med. 2019 Aug 8;381(6):509-519. doi: 10.1056/NEJMoa1903212. Epub 2019 Jun 14.
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Therapeutic strategies for sickle cell disease: towards a multi-agent approach.镰状细胞病的治疗策略：迈向多药物联合治疗方法。
Nat Rev Drug Discov. 2019 Feb;18(2):139-158. doi: 10.1038/s41573-018-0003-2.
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Emerging Genetic Therapy for Sickle Cell Disease.新兴的镰状细胞病基因治疗。
Annu Rev Med. 2019 Jan 27;70:257-271. doi: 10.1146/annurev-med-041817-125507. Epub 2018 Oct 24.
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Recent progress in understanding and manipulating haemoglobin switching for the haemoglobinopathies.血红蛋白病中血红蛋白开关理解和操作的最新进展。
Br J Haematol. 2018 Mar;180(5):630-643. doi: 10.1111/bjh.15038. Epub 2017 Nov 28.
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CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells.CRISPR/Cas9对人类造血干细胞β-珠蛋白基因的靶向作用。
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