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Search results (380)

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Systemic Inflammation Modulates Clearance and Drives Extra-Hepatic Distribution of Extracellular Vesicles.

Pavlova S. et al, (2026), J Extracell Vesicles, 15

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

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

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

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

Targeted BDNF upregulation via upstream open reading frame disruption.

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

Timing matters: exon skipping therapy is most effective when initiated early in a mouse model of Duchenne muscular dystrophy

Stenler S. et al, (2025)

The CTDP1 Founder Variant in CCFDN: Insights into Pathogenesis, Phenotypic Spectrum and Therapeutic Approaches.

Sabau IM. et al, (2025), Int J Mol Sci, 27

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

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

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

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

Biophysical and biological properties of splice-switching oligonucleotides and click conjugates containing LNA-phosphothiotriester linkages.

Dhara D. et al, (2025), Nucleic Acids Res, 53

Synthesis, Biological Activity, and Molecular Dynamics Simulations of LNA-Charge Neutral Linkages for Enhanced Splice-Switching Antisense Oligonucleotides.

Kennett A. et al, (2025), Angew Chem Int Ed Engl, 64

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

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

miR-107 represses DMPK and is sequestered by CUG repeats triggering the MSI2/miR-7 pathogenesis axis in myotonic dystrophy.

Moreno N. et al, (2025), Mol Ther Nucleic Acids, 36

Galectin-3 induces neurodevelopmental apical-basal polarity and regulates gyrification.

Soares LC. et al, (2025), Sci Adv, 11

Myonuclear domain-associated and central nucleation-dependent spatial restriction of dystrophin protein expression in a novel DMD mouse model

Chwalenia K. et al, (2025)

Cell-penetrating peptide-conjugated, splice-switching oligonucleotides mitigate the phenotype in BTK/Tec double deficient X-linked agammaglobulinemia model.

Bestas B. et al, (2025), RSC Chem Biol, 6, 761 - 771

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