Imagine CRISPR cures.
作者信息
Urnov Fyodor D
机构信息
Professor, Molecular and Cell Biology Department, Scientific Director, Technology and Translation, Innovative Genomics Institute, UC Berkeley, 2151 Berkeley Way, Berkeley, CA 94720, USA.
出版信息
Mol Ther. 2021 Nov 3;29(11):3103-3106. doi: 10.1016/j.ymthe.2021.10.019. Epub 2021 Oct 28.
Abstract
摘要
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N Engl J Med. 2021 Aug 5;385(6):493-502. doi: 10.1056/NEJMoa2107454. Epub 2021 Jun 26.
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Rapid Sequencing-Based Diagnosis of Thiamine Metabolism Dysfunction Syndrome.基于快速测序的硫胺素代谢功能障碍综合征诊断
N Engl J Med. 2021 Jun 3;384(22):2159-2161. doi: 10.1056/NEJMc2100365.
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Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency.腺苷脱氨酶缺乏症的自体体外慢病毒基因治疗。
N Engl J Med. 2021 May 27;384(21):2002-2013. doi: 10.1056/NEJMoa2027675. Epub 2021 May 11.
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The NIH Somatic Cell Genome Editing program.美国国立卫生研究院体细胞基因组编辑计划。
Nature. 2021 Apr;592(7853):195-204. doi: 10.1038/s41586-021-03191-1. Epub 2021 Apr 7.
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The Cas9 Hammer-and Sickle: A Challenge for Genome Editors.《Cas9 双刃剑：基因组编辑的挑战》
CRISPR J. 2021 Feb;4(1):6-13. doi: 10.1089/crispr.2021.29120.fur.
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CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia.CRISPR-Cas9 基因编辑治疗镰状细胞病和 β-地中海贫血。
N Engl J Med. 2021 Jan 21;384(3):252-260. doi: 10.1056/NEJMoa2031054. Epub 2020 Dec 5.
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Inherited SLP76 deficiency in humans causes severe combined immunodeficiency, neutrophil and platelet defects.人类遗传性 SLP76 缺乏导致严重联合免疫缺陷、中性粒细胞和血小板缺陷。
J Exp Med. 2021 Mar 1;218(3). doi: 10.1084/jem.20201062.
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CRISPR-CasΦ from huge phages is a hypercompact genome editor.来自巨型噬菌体的 CRISPR-CasΦ 是一种超紧凑型基因组编辑工具。
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Genome editing with CRISPR-Cas nucleases, base editors, transposases and prime editors.利用 CRISPR-Cas 核酸酶、碱基编辑器、转座酶和 Prime 编辑器进行基因组编辑。
Nat Biotechnol. 2020 Jul;38(7):824-844. doi: 10.1038/s41587-020-0561-9. Epub 2020 Jun 22.
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Unconstrained genome targeting with near-PAMless engineered CRISPR-Cas9 variants.无约束基因组靶向的近无 PAM 工程化 CRISPR-Cas9 变体。
Science. 2020 Apr 17;368(6488):290-296. doi: 10.1126/science.aba8853. Epub 2020 Mar 26.
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