Article

Evaluating exon-skipping therapies targeting the central nervous system in Duchenne muscular dystrophy using high-resolution spatial transcriptomics

2025-10-20

Abstract excerpt

Duchenne muscular dystrophy (DMD) is marked by progressive muscle degeneration due to dystrophin deficiency. Despite cognitive impairments in up to one–third of patients, central nervous system (CNS)–targeted dystrophin restoration remains relatively underexplored compared to muscle-focused therapies. Since dystrophin is expressed in the brain during development and postnatally, it is important to assess which asp...

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Literature Corpus work: c372e444-b4d3-5343-83f2-45a8b659704e
DOI: 10.1101/2025.10.20.683239

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