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Search results (375)

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Targeted BDNF upregulation via upstream open reading frame disruption

Feng N. et al, (2026), Molecular Therapy, 34, 1652 - 1671

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

Woffindale C. et al, (2026), Molecular therapy. 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)

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), Journal of Muscle Research and Cell Motility, 46, 293 - 300

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

Sabau IM. et al, (2025), International journal of molecular sciences, 27

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

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

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

Kennett A. et al, (2025), Angewandte Chemie (International ed. in English), 64

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

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

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

Soares LC. et al, (2025), Science Advances, 11

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), Molecular therapy. Nucleic acids, 36

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 chemical biology, 6, 761 - 771

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

Liang X. et al, (2025), Nature communications, 16

Targeted BDNF upregulation via upstream open reading frame disruption

Feng N. et al, (2025)

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