CRISPR for Neuromuscular Disorders: Gene Editing and Beyond

Courtney S. Young; April D. Pyle; Melissa J. Spencer

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CRISPR for Neuromuscular Disorders: Gene Editing and Beyond

机译：神经肌肉疾病的CRISPR：基因编辑及超越

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摘要

This is a review describing advances in CRISPR/Cas-mediated therapies for neuromuscular disorders (NMDs). We explore both CRISPR-mediated editing and dead Cas approaches as potential therapeutic strategies for multiple NMDs. Last, therapeutic considerations, including delivery and off-target effects, are also discussed.

机译：本文综述了CRISPR/Cas介导的神经肌肉疾病（NMD）治疗的进展。我们探讨了CRISPR介导的编辑和死亡Cas方法作为多种NMD的潜在治疗策略。最后，还讨论了治疗注意事项，包括给药和靶向外效应。

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来源
《Physiology》 |2019年第9期|共13页
作者
Courtney S. Young; April D. Pyle; Melissa J. Spencer;
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作者单位

Department of Neurology University of California Los Angeles California;

Center for Duchenne Muscular Dystrophy at UCLA University of California Los Angeles California;

Center for Duchenne Muscular Dystrophy at UCLA University of California Los Angeles California;

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原文格式 PDF
正文语种 eng
中图分类生理学;
关键词
CRISPR/Cas; gene editing; neuromuscular disorders; muscular dystrophy; gene therapy;

机译：CRISPR / CAS;基因编辑;神经肌肉障碍;肌营养不良;基因治疗;
入库时间 2022-08-20 19:00:48

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1. Multiplex gene editing in rice with simplified CRISPR-Cpf1 and CRISPR-Cas9 systemsFA [J] . Mugui Wang, Yanfei Mao, Yuming Lu, 植物学报（英文版） . 2018,第008期
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3. Comparing successful gene knock-in efficiencies of CRISPR/Cas9 with ZFNs and TALENs gene editing systems in bovine and dairy goat fetal fibroblasts [J] . LIU Hui12, LIU Chang12, ZHAO Yu-hang12, 农业科学学报：英文版 . 2018,第002期
4. Design of synthetic materials for intracellular delivery of RNAs： From siRNA-mediated gene silencing to CRISPR/Cas gene editing [J] . Jason B. Miller, Daniel J. Siegwart 纳米研究：英文版 . 2018,第010期
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机译：神经肌肉疾病的CRISPR：基因编辑及超越
7. Gene editing using CRISPR/Cas9 in neuromuscular disorders [J] . Gonorazky H., Maani N., Khattak S., Neuromuscular disorders: NMD . 2016,第Suppla2期

机译：使用CRISPR / Cas9进行神经肌肉疾病的基因编辑
8. Gene Therapy 2.0: CRISPR/Cas9 genome editing provides novel therapeutic avenues for neuromuscular disorders [J] . Cohn R. Neuromuscular disorders: NMD . 2016,第Suppla2期

机译：基因治疗2.0：CRISPR / Cas9基因组编辑为神经肌肉疾病提供了新颖的治疗途径
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机译：羧基化的支化聚（β-氨基酯）纳米粒子可实现强大的细胞内蛋白质递送和CRISPR / Cas9基因编辑
10. Enhancing the Efficiency of CRISPR/Cas9 Precise Gene Editing for Cystic Fibrosis Gene Therapy [D] . Seigel, Kyle E. 2018

机译：增强CRISPR / Cas9精确基因编辑对囊性纤维化基因治疗的效率
11. CRISPR-cas gene-editing as plausible treatment of neuromuscular and nucleotide-repeat-expansion diseases: A systematic review [O] . Haris Babačić, Aditi Mehta, Olivia Merkel, -1

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12. CRISPR Gene-Editing Models Geared Toward Therapy for Hereditary and Developmental Neurological Disorders [O] . Poh Kuan Wong, Fook Choe Cheah, Saiful Effendi Syafruddin, 2021

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机译：国家咨询神经和交流障碍和卒中委员会关于惊厥和神经肌肉疾病专家组的报告。第4卷

CRISPR for Neuromuscular Disorders: Gene Editing and Beyond

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