作者更正：在杜兴氏肌营养不良症的一种新小鼠模型中，细胞介导的外显子跳跃使肌营养不良蛋白表达和肌肉功能正常化。
Author Correction: Cell-mediated exon skipping normalizes dystrophin expression and muscle function in a new mouse model of Duchenne Muscular Dystrophy.
作者信息
Galli Francesco, Bragg Laricia, Rossi Maira, Proietti Daisy, Perani Laura, Bacigaluppi Marco, Tonlorenzi Rossana, Sibanda Tendai, Caffarini Miriam, Talapatra Avraneel, Santoleri Sabrina, Meregalli Mirella, Bano-Otalora Beatriz, Bigot Anne, Bozzoni Irene, Bonini Chiara, Mouly Vincent, Torrente Yvan, Cossu Giulio
机构信息
Division of Cell Matrix Biology & Regenerative Medicine, Faculty of Biology, Medicine and Health, University of Manchester, Manchester, UK.
Institute of Experimental Neurology, Division of Neurosciences, IRCCS San Raffaele Scientific Institute, Milan, Italy.
出版信息
EMBO Mol Med. 2024 Oct;16(10):2654. doi: 10.1038/s44321-024-00134-x.
Abstract
[Image: see text]
摘要
[图片：见正文]
相似文献
1
Author Correction: Cell-mediated exon skipping normalizes dystrophin expression and muscle function in a new mouse model of Duchenne Muscular Dystrophy.作者更正：在杜兴氏肌营养不良症的一种新小鼠模型中，细胞介导的外显子跳跃使肌营养不良蛋白表达和肌肉功能正常化。
EMBO Mol Med. 2024 Oct;16(10):2654. doi: 10.1038/s44321-024-00134-x.
2
Cell-mediated exon skipping normalizes dystrophin expression and muscle function in a new mouse model of Duchenne Muscular Dystrophy.细胞介导的外显子跳跃可使一种新型杜氏肌营养不良症小鼠模型中的抗肌萎缩蛋白表达和肌肉功能正常化。
EMBO Mol Med. 2024 Apr;16(4):927-944. doi: 10.1038/s44321-024-00031-3. Epub 2024 Mar 4.
3
Targeted exon skipping as a potential gene correction therapy for Duchenne muscular dystrophy.靶向外显子跳跃作为杜氏肌营养不良症的一种潜在基因校正疗法。
Neuromuscul Disord. 2002 Oct;12 Suppl 1:S71-7. doi: 10.1016/s0960-8966(02)00086-x.
4
Dantrolene enhances antisense-mediated exon skipping in human and mouse models of Duchenne muscular dystrophy.丹曲林增强了杜氏肌营养不良症人类和小鼠模型中反义介导的外显子跳跃。
Sci Transl Med. 2012 Dec 12;4(164):164ra160. doi: 10.1126/scitranslmed.3005054.
5
Efficacy of Multi-exon Skipping Treatment in Duchenne Muscular Dystrophy Dog Model Neonates.多外显子跳跃治疗在杜氏肌营养不良症犬模型新生儿中的疗效。
Mol Ther. 2019 Jan 2;27(1):76-86. doi: 10.1016/j.ymthe.2018.10.011. Epub 2018 Oct 19.
6
Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse.AAV9外显子跳跃载体的全身递送显著改善或预防了Dup2小鼠的杜氏肌营养不良症特征。
Mol Ther Methods Clin Dev. 2022 Jul 11;26:279-293. doi: 10.1016/j.omtm.2022.07.005. eCollection 2022 Sep 8.
7
Dystrophin quantification and clinical correlations in Becker muscular dystrophy: implications for clinical trials.Becker 型肌营养不良症中抗肌萎缩蛋白的定量检测及其与临床的相关性：对临床试验的影响。
Brain. 2011 Dec;134(Pt 12):3547-59. doi: 10.1093/brain/awr291. Epub 2011 Nov 18.
8
A duchenne muscular dystrophy gene hot spot mutation in dystrophin-deficient cavalier king charles spaniels is amenable to exon 51 skipping.肌营养不良蛋白缺乏型小型骑士查理王小猎犬存在杜兴氏肌营养不良症基因热点突变，可采用外显子 51 跳跃法进行治疗。
PLoS One. 2010 Jan 13;5(1):e8647. doi: 10.1371/journal.pone.0008647.
9
Autoimmune response and its long-term consequences after exon-skipping therapy in a Duchenne muscular dystrophy mouse model.跳跃外显子治疗杜氏肌营养不良症小鼠模型后的自身免疫反应及其长期后果。
J Pathol. 2019 Nov;249(3):271-273. doi: 10.1002/path.5327. Epub 2019 Aug 31.
10
Next Generation Exon 51 Skipping Antisense Oligonucleotides for Duchenne Muscular Dystrophy.用于杜氏肌营养不良症的下一代外显子 51 跳跃反义寡核苷酸。
Nucleic Acid Ther. 2023 Jun;33(3):193-208. doi: 10.1089/nat.2022.0063. Epub 2023 Apr 10.
引用本文的文献
1
Transcriptional adaptation upregulates utrophin in Duchenne muscular dystrophy.转录适应性上调杜氏肌营养不良症中的抗肌萎缩蛋白。
Nature. 2025 Mar;639(8054):493-502. doi: 10.1038/s41586-024-08539-x. Epub 2025 Feb 12.
Zotero 插件