Author

P. Spitali

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Open access Jul 2026

Evaluating exon skipping in the central nervous system in Duchenne muscular dystrophy using spatial transcriptomics

Evidence of isoform restoration, immune activation following treatment, and a framework to evaluate exon-skipping therapies in the CNS using spatial transcriptomics are presented.

Qirong Mao, A. Ahmadi, S. D. de Vries et al. · iScience · 0 citations

Open access Aug 2026

Givinostat enhances antisense oligonucleotide efficacy in the mdx52 model of Duchenne muscular dystrophy

Givinostat significantly improved exon skipping and dystrophin restoration in human DMD myoblasts carrying an exon 52 deletion, and combining givinostat with ASOs to enhance dystrophin restoration and muscle preservation in DMD.

Xaysongkhame Phongsavanh, C. Gastaldi, Astrid Mottais et al. · Molecular Therapy: Nucleic A... · 0 citations