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Updated: May 28, 2025

Multi-exon Skipping Using Cocktail Antisense Oligonucleotides in the Canine X-linked Muscular Dystrophy
10:30

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Published on: May 24, 2016

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对异临线病变的前离子跳转适用性的分析

Jamie Leckie1, Sebastian Hernandez Rodriguez1, Martin Krahn2,3

  • 1Department of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, AB T6G 2H7, Canada.

Cells
|February 12, 2025
PubMed
概括

反感性寡核酸 (ASO) 通过向DYSF基因,为异性ferlinopathies提供有前途的外因子跳转策略. 这项研究确定了61种策略,有可能解决90%的致病变体,指导未来的治疗开发.

关键词:
这是一种反感性寡核酸 (ASO).适用性 适用性 适用性这种疾病被称为dysferlinopathy.突出跳过的突出跳过

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Direct Reprogramming of Human Fibroblasts into Myoblasts to Investigate Therapies for Neuromuscular Disorders10:28

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