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Search results (378)

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

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

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

Targeted BDNF upregulation via upstream open reading frame disruption.

Feng N. et al, (2025), Mol Ther

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

Engineering of extracellular vesicles for efficient intracellular delivery of multimodal therapeutics including genome editors.

Liang X. et al, (2025), Nat Commun, 16

uORF-targeting steric block antisense oligonucleotides do not reproducibly increase RNASEH1 expression.

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

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