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Base editing: advances and therapeutic opportunities.碱基编辑：进展与治疗机遇。
Nat Rev Drug Discov. 2020 Dec;19(12):839-859. doi: 10.1038/s41573-020-0084-6. Epub 2020 Oct 19.
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CRISPR technologies for the treatment of Duchenne muscular dystrophy.CRISPR 技术治疗杜氏肌营养不良症。
Mol Ther. 2021 Nov 3;29(11):3179-3191. doi: 10.1016/j.ymthe.2021.04.002. Epub 2021 Apr 3.
3
CRISPR/Cas correction of muscular dystrophies.CRISPR/Cas 纠正肌肉疾病。
Exp Cell Res. 2021 Nov 1;408(1):112844. doi: 10.1016/j.yexcr.2021.112844. Epub 2021 Sep 25.
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In Vivo Genome Editing Restores Dystrophin Expression and Cardiac Function in Dystrophic Mice.体内基因组编辑可恢复营养不良小鼠的肌营养不良蛋白表达和心脏功能。
Circ Res. 2017 Sep 29;121(8):923-929. doi: 10.1161/CIRCRESAHA.117.310996. Epub 2017 Aug 8.
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Molecular correction of Duchenne muscular dystrophy by splice modulation and gene editing.通过剪接调控和基因编辑实现杜氏肌营养不良症的分子矫正。
RNA Biol. 2021 Jul;18(7):1048-1062. doi: 10.1080/15476286.2021.1874161. Epub 2021 Jan 20.
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CRISPR-Cas9 Gene Therapy for Duchenne Muscular Dystrophy.CRISPR-Cas9 基因治疗杜氏肌营养不良症。
Neurotherapeutics. 2022 Apr;19(3):931-941. doi: 10.1007/s13311-022-01197-9. Epub 2022 Feb 14.
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Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy.AAV-CRISPR 基因组编辑治疗杜氏肌营养不良症的长期评估。
Nat Med. 2019 Mar;25(3):427-432. doi: 10.1038/s41591-019-0344-3. Epub 2019 Feb 18.
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CRISPR-Based Therapeutic Gene Editing for Duchenne Muscular Dystrophy: Advances, Challenges and Perspectives.基于 CRISPR 的杜氏肌营养不良症治疗性基因编辑：进展、挑战与展望。
Cells. 2022 Sep 22;11(19):2964. doi: 10.3390/cells11192964.
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Toward the correction of muscular dystrophy by gene editing.通过基因编辑纠正肌肉营养不良症。
Proc Natl Acad Sci U S A. 2021 Jun 1;118(22). doi: 10.1073/pnas.2004840117. Epub 2021 Apr 30.
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Correction of muscular dystrophies by CRISPR gene editing.通过 CRISPR 基因编辑纠正肌肉疾病。
J Clin Invest. 2020 Jun 1;130(6):2766-2776. doi: 10.1172/JCI136873.
引用本文的文献
1
Functional phenotyping of genomic variants using joint multiomic single-cell DNA-RNA sequencing.使用联合多组学单细胞DNA-RNA测序对基因组变异进行功能表型分析。
Nat Methods. 2025 Sep 1. doi: 10.1038/s41592-025-02805-0.
2
CRISPR tools for T cells: targeting the genome, epigenome, and transcriptome.用于T细胞的CRISPR工具：靶向基因组、表观基因组和转录组。
Trends Cancer. 2025 Aug 28. doi: 10.1016/j.trecan.2025.08.001.
3
Beyond transfusions and transplants: genomic innovations rewriting the narrative of thalassemia.超越输血和移植：基因组创新改写地中海贫血的故事
Ann Hematol. 2025 Aug 16. doi: 10.1007/s00277-025-06548-y.
4
Viral and nonviral nanocarriers for CRISPR-based gene editing.用于基于CRISPR的基因编辑的病毒和非病毒纳米载体。
Nano Res. 2024 Oct;17(10):8904-8925. doi: 10.1007/s12274-024-6748-5. Epub 2024 Jun 20.
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Genome Editing Breeding with CRISPR-Cas Nucleases, Base Editors, and Prime Editors.基于CRISPR-Cas核酸酶、碱基编辑器和引导编辑器的基因组编辑育种
Animals (Basel). 2025 Jul 22;15(15):2161. doi: 10.3390/ani15152161.
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Gene editing for collagen disorders: current advances and future perspectives.用于胶原蛋白疾病的基因编辑：当前进展与未来展望。
Gene Ther. 2025 Aug 11. doi: 10.1038/s41434-025-00560-7.
7
Tracing the development of CAR-T cell design: from concept to next-generation platforms.追溯嵌合抗原受体T细胞（CAR-T）设计的发展：从概念到下一代平台。
Front Immunol. 2025 Jul 17;16:1615212. doi: 10.3389/fimmu.2025.1615212. eCollection 2025.
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Bioengineering Outer-Membrane Vesicles for Vaccine Development: Strategies, Advances, and Perspectives.用于疫苗开发的生物工程外膜囊泡：策略、进展与展望
Vaccines (Basel). 2025 Jul 20;13(7):767. doi: 10.3390/vaccines13070767.
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Towards improved fine-mapping of candidate causal variants.迈向对候选因果变异更精细的定位。
Nat Rev Genet. 2025 Jul 28. doi: 10.1038/s41576-025-00869-4.
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Removal of promoter CpG methylation by epigenome editing reverses HBG silencing.通过表观基因组编辑去除启动子CpG甲基化可逆转HBG沉默。
Nat Commun. 2025 Jul 27;16(1):6919. doi: 10.1038/s41467-025-62177-z.
本文引用的文献
1
Engineered materials for in vivo delivery of genome-editing machinery.用于体内递送基因组编辑机制的工程材料。
Nat Rev Mater. 2019 Nov;4:726-737. doi: 10.1038/s41578-019-0145-9. Epub 2019 Oct 4.
2
Massively parallel assessment of human variants with base editor screens.基于碱基编辑器的高通量人类变异评估。
Cell. 2021 Feb 18;184(4):1064-1080.e20. doi: 10.1016/j.cell.2021.01.012.
3
PrimeDesign software for rapid and simplified design of prime editing guide RNAs.PrimeDesign 软件可快速简化向导 RNA 设计。
Nat Commun. 2021 Feb 15;12(1):1034. doi: 10.1038/s41467-021-21337-7.
4
multicrispr: gRNA design for prime editing and parallel targeting of thousands of targets.multicrispr：用于 prime editing 和数千个靶点并行靶向的 gRNA 设计。
Life Sci Alliance. 2020 Sep 9;3(11). doi: 10.26508/lsa.202000757. Print 2020 Nov.
5
CRISPR C-to-G base editors for inducing targeted DNA transversions in human cells.CRISPR C 到 G 碱基编辑器在人类细胞中诱导靶向 DNA 颠换。
Nat Biotechnol. 2021 Jan;39(1):41-46. doi: 10.1038/s41587-020-0609-x. Epub 2020 Jul 20.
6
Glycosylase base editors enable C-to-A and C-to-G base changes.糖苷酶碱基编辑器可实现 C 到 A 和 C 到 G 的碱基变化。
Nat Biotechnol. 2021 Jan;39(1):35-40. doi: 10.1038/s41587-020-0592-2. Epub 2020 Jul 20.
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A bacterial cytidine deaminase toxin enables CRISPR-free mitochondrial base editing.一种细菌胞嘧啶脱氨酶毒素可实现无 CRISPR 的线粒体碱基编辑。
Nature. 2020 Jul;583(7817):631-637. doi: 10.1038/s41586-020-2477-4. Epub 2020 Jul 8.
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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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Determinants of Base Editing Outcomes from Target Library Analysis and Machine Learning.从目标文库分析和机器学习角度看碱基编辑结果的决定因素。
Cell. 2020 Jul 23;182(2):463-480.e30. doi: 10.1016/j.cell.2020.05.037. Epub 2020 Jun 12.
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In vivo base editing restores sensory transduction and transiently improves auditory function in a mouse model of recessive deafness.在体内进行碱基编辑可恢复隐性耳聋小鼠模型中的感觉转导，并短暂改善听觉功能。
Sci Transl Med. 2020 Jun 3;12(546). doi: 10.1126/scitranslmed.aay9101.
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