Combined 20-Hydroxyecdysone and Antisense-Mediated Exon Skipping Improve Functional Outcomes in a Mouse Model of Duchenne Muscular Dystrophy.

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Tác giả: Mathilde Blitek, Mathilde Doisy, Luis Garcia, Cécile Gastaldi, Aurélie Goyenvalle, Olivier Le Coz, Thomas Tensorer

Ngôn ngữ: eng

Ký hiệu phân loại:

Thông tin xuất bản: United States : Nucleic acid therapeutics , 2025

Mô tả vật lý:

Bộ sưu tập: NCBI

ID: 181151

Duchenne muscular dystrophy (DMD) is a severe X-linked disorder caused by mutations in the DMD gene, resulting in a lack of dystrophin protein. This leads to progressive muscle wasting, cardiac and respiratory dysfunction, and premature death. Antisense oligonucleotide (ASO)-based therapies represent a promising approach to treating DMD, with several already approved by the FDA. However, the levels of dystrophin restoration achieved in clinical trials are often insufficient for meaningful therapeutic impact, highlighting the urgent need to enhance ASO efficacy. One potential strategy is to improve muscle pathophysiology, which is compromised in DMD due to cycles of necrosis and regeneration, chronic inflammation, and fibrotic and adipose tissue replacement. These disease characteristics may limit ASO efficiency. In this study, we evaluated the combination of tricyclo-DNA-ASO targeting the
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