Toward life without sickle cell disease.
作者信息
Davé Utpal P, Bungert Jörg
机构信息
Department of Medicine, Division of Hematology/Oncology, Indiana University School of Medicine, Roudebush VA Medical Center, and IU Simon Comprehensive Cancer Center, Indianapolis, IN, USA.
Department of Biochemistry and Molecular Biology, UF Health Cancer Center, Center for Epigenetics, Genetics Institute, Powell Gene Therapy Center, University of Florida, Gainesville, FL 32610, USA.
出版信息
Mol Ther. 2024 Oct 2;32(10):3197-3198. doi: 10.1016/j.ymthe.2024.09.004. Epub 2024 Sep 17.
Abstract
摘要
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本文引用的文献
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Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34 cell therapy to induce fetal hemoglobin for sickle cell disease.新型 Cas9 基因编辑自体 CD34 细胞疗法诱导镰状细胞病胎儿血红蛋白的开发和 IND 支持研究。
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In utero delivery of targeted ionizable lipid nanoparticles facilitates in vivo gene editing of hematopoietic stem cells.经子宫内递送达靶可离子化脂质纳米颗粒促进了造血干细胞的体内基因编辑。
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Mol Cell. 2023 Oct 5;83(19):3533-3545.e5. doi: 10.1016/j.molcel.2023.09.008.
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Genotoxic effects of base and prime editing in human hematopoietic stem cells.碱基编辑和先导编辑在人造血干细胞中的遗传毒性效应。
Nat Biotechnol. 2024 Jun;42(6):877-891. doi: 10.1038/s41587-023-01915-4. Epub 2023 Sep 7.
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Sickle cell disease.镰状细胞病。
Nat Rev Dis Primers. 2018 Mar 15;4:18010. doi: 10.1038/nrdp.2018.10.
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Transcription factors LRF and BCL11A independently repress expression of fetal hemoglobin.转录因子LRF和BCL11A分别抑制胎儿血红蛋白的表达。
Science. 2016 Jan 15;351(6270):285-9. doi: 10.1126/science.aad3312.
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