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Myonuclear Domain-Associated and Central Nucleation-Dependent Spatial Restriction of Dystrophin Protein Expression.

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Chwalenia K. et al, (2026), J Cachexia Sarcopenia Muscle, 17

Design, validation, and functional impact of oligonucleotides for multigene silencing in Alzheimer's disease.

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Woffindale C. et al, (2026), Mol Ther Nucleic Acids, 37

Enhanced muscle uptake of chemically optimized miR-23b antisense oligonucleotides as lead compounds for myotonic dystrophy type 1.

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González-Martínez I. et al, (2026), Am J Hum Genet, 113, 529 - 547

Targeted BDNF upregulation via upstream open reading frame disruption.

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Feng N. et al, (2026), Mol Ther, 34, 1652 - 1671

Exon skipping peptide-conjugated morpholinos downregulate dynamin 2 to rescue centronuclear myopathy.

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Moschovaki-Filippidou F. et al, (2025), Brain, 148, 4495 - 4507

Progress and prospects in antisense oligonucleotide-mediated exon skipping therapies for Duchenne muscular dystrophy.

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Chwalenia K. et al, (2025), J Muscle Res Cell Motil, 46, 293 - 300

AAV microdystrophin gene replacement therapy for Duchenne muscular dystrophy: progress and prospects.

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Chwalenia K. et al, (2025), Gene Ther, 32, 447 - 461

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