手术刀还是约束衣：用于治疗肌肉萎缩症的CRISPR/Cas9方法
Scalpel or Straitjacket: CRISPR/Cas9 Approaches for Muscular Dystrophies.
作者信息
Himeda Charis L, Jones Takako I, Jones Peter L
机构信息
The Department of Cell and Developmental Biology, University of Massachusetts Medical School, Worcester, MA, 01655, USA.
The Department of Cell and Developmental Biology, University of Massachusetts Medical School, Worcester, MA, 01655, USA.
出版信息
Trends Pharmacol Sci. 2016 Apr;37(4):249-251. doi: 10.1016/j.tips.2016.02.001. Epub 2016 Feb 22.
Abstract
Versatility of CRISPR/Cas9-based platforms makes them promising tools for the correction of diverse genetic/epigenetic disorders. Here we contrast the use of these genome editing tools in two myopathies with very different molecular origins: Duchenne muscular dystrophy, a monogenetic disease, and facioscapulohumeral muscular dystrophy, an epigenetic disorder with unique therapeutic challenges.
摘要
基于CRISPR/Cas9的平台的多功能性使其成为纠正各种遗传/表观遗传疾病的有前景的工具。在这里，我们对比了这些基因组编辑工具在两种分子起源截然不同的肌病中的应用：杜氏肌营养不良症，一种单基因疾病；以及面肩肱型肌营养不良症，一种具有独特治疗挑战的表观遗传疾病。
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本文引用的文献
1
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Mol Ther. 2016 Mar;24(3):430-46. doi: 10.1038/mt.2016.10. Epub 2016 Jan 12.
2
In vivo gene editing in dystrophic mouse muscle and muscle stem cells.营养不良小鼠肌肉和肌肉干细胞中的体内基因编辑。
Science. 2016 Jan 22;351(6271):407-411. doi: 10.1126/science.aad5177. Epub 2015 Dec 31.
3
In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy.体内基因组编辑改善了杜兴氏肌肉营养不良小鼠模型的肌肉功能。
Science. 2016 Jan 22;351(6271):403-7. doi: 10.1126/science.aad5143. Epub 2015 Dec 31.
4
Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy.产后基因编辑可部分恢复肌营养不良小鼠模型中的肌营养不良蛋白表达。
Science. 2016 Jan 22;351(6271):400-3. doi: 10.1126/science.aad5725. Epub 2015 Dec 31.
5
Spell Checking Nature: Versatility of CRISPR/Cas9 for Developing Treatments for Inherited Disorders.拼写检查的本质：CRISPR/Cas9在开发遗传性疾病治疗方法中的多功能性。
Am J Hum Genet. 2016 Jan 7;98(1):90-101. doi: 10.1016/j.ajhg.2015.11.012. Epub 2015 Dec 10.
6
CRISPR/dCas9-mediated Transcriptional Inhibition Ameliorates the Epigenetic Dysregulation at D4Z4 and Represses DUX4-fl in FSH Muscular Dystrophy.CRISPR/dCas9介导的转录抑制改善了D4Z4处的表观遗传失调，并抑制了FSH型肌营养不良症中的DUX4-fl。
Mol Ther. 2016 Mar;24(3):527-35. doi: 10.1038/mt.2015.200. Epub 2015 Nov 3.
7
Trans-spliced Cas9 allows cleavage of HBB and CCR5 genes in human cells using compact expression cassettes.反式剪接的Cas9可利用紧凑表达盒在人类细胞中切割HBB和CCR5基因。
Sci Rep. 2015 Jul 1;5:10777. doi: 10.1038/srep10777.
8
High-Throughput Silencing Using the CRISPR-Cas9 System: A Review of the Benefits and Challenges.使用CRISPR-Cas9系统的高通量基因沉默：益处与挑战综述
J Biomol Screen. 2015 Sep;20(8):1027-39. doi: 10.1177/1087057115587916. Epub 2015 May 22.
9
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