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。
相似文献
1
Scalpel or Straitjacket: CRISPR/Cas9 Approaches for Muscular Dystrophies.
Trends Pharmacol Sci. 2016 Apr;37(4):249-251. doi: 10.1016/j.tips.2016.02.001. Epub 2016 Feb 22.
2
Gene Editing for Duchenne Muscular Dystrophy Using the CRISPR/Cas9 Technology: The Importance of Fine-tuning the Approach.
Mol Ther. 2016 Nov;24(11):1888-1889. doi: 10.1038/mt.2016.191.
3
CRISPR-Based Gene Therapies: From Preclinical to Clinical Treatments.
Cells. 2024 May 8;13(10):800. doi: 10.3390/cells13100800.
4
Advances in CRISPR/Cas9 Genome Editing for the Treatment of Muscular Dystrophies.
Hum Gene Ther. 2023 May;34(9-10):388-403. doi: 10.1089/hum.2023.059.
5
Applying genome-wide CRISPR-Cas9 screens for therapeutic discovery in facioscapulohumeral muscular dystrophy.
Sci Transl Med. 2020 Mar 25;12(536). doi: 10.1126/scitranslmed.aay0271.
6
CRISPR-Cas9 Gene Therapy for Duchenne Muscular Dystrophy.
Neurotherapeutics. 2022 Apr;19(3):931-941. doi: 10.1007/s13311-022-01197-9. Epub 2022 Feb 14.
7
A novel rabbit model of Duchenne muscular dystrophy generated by CRISPR/Cas9.
Dis Model Mech. 2018 Jun 4;11(6):dmm032201. doi: 10.1242/dmm.032201.
8
CRISPR technologies for the treatment of Duchenne muscular dystrophy.
Mol Ther. 2021 Nov 3;29(11):3179-3191. doi: 10.1016/j.ymthe.2021.04.002. Epub 2021 Apr 3.
9
The AAV-mediated and RNA-guided CRISPR/Cas9 system for gene therapy of DMD and BMD.
Brain Dev. 2017 Aug;39(7):547-556. doi: 10.1016/j.braindev.2017.03.024. Epub 2017 Apr 5.
10
CRISPR/Cas correction of muscular dystrophies.
Exp Cell Res. 2021 Nov 1;408(1):112844. doi: 10.1016/j.yexcr.2021.112844. Epub 2021 Sep 25.
引用本文的文献
1
FSHD Therapeutic Strategies: What Will It Take to Get to Clinic?
J Pers Med. 2022 May 25;12(6):865. doi: 10.3390/jpm12060865.
2
Targeted epigenetic repression by CRISPR/dSaCas9 suppresses pathogenic expression in FSHD.
Mol Ther Methods Clin Dev. 2020 Dec 10;20:298-311. doi: 10.1016/j.omtm.2020.12.001. eCollection 2021 Mar 12.
3
Therapeutic Approaches in Facioscapulohumeral Muscular Dystrophy.
Trends Mol Med. 2021 Feb;27(2):123-137. doi: 10.1016/j.molmed.2020.09.008. Epub 2020 Oct 19.
4
Antisense Oligonucleotides Used to Target the DUX4 mRNA as Therapeutic Approaches in FaciosScapuloHumeral Muscular Dystrophy (FSHD).
Genes (Basel). 2017 Mar 3;8(3):93. doi: 10.3390/genes8030093.
本文引用的文献
1
Genome-editing Technologies for Gene and Cell Therapy.
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.
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.
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.
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.
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.
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.
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.
J Biomol Screen. 2015 Sep;20(8):1027-39. doi: 10.1177/1087057115587916. Epub 2015 May 22.
9
Individual epigenetic status of the pathogenic D4Z4 macrosatellite correlates with disease in facioscapulohumeral muscular dystrophy.
Clin Epigenetics. 2015 Mar 29;7(1):37. doi: 10.1186/s13148-015-0072-6. eCollection 2015.
10
In vivo genome editing using Staphylococcus aureus Cas9.
Nature. 2015 Apr 9;520(7546):186-91. doi: 10.1038/nature14299. Epub 2015 Apr 1.
Zotero 插件