Last Updated: 09/26/2026
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Found 221 publications
Accurately modeling RNase H-mediated antisense oligonucleotide efficacy.
Journal: Molecular therapy. Nucleic acids
Published: January 21, 2026
Timing matters: Exon skipping therapy is most effective when initiated early in a mouse model of Duchenne muscular dystrophy.
Journal: Molecular therapy. Nucleic acids
Published: January 12, 2026
The CTDP1 Founder Variant in CCFDN: Insights into Pathogenesis, Phenotypic Spectrum and Therapeutic Approaches.
Journal: International journal of molecular sciences
Published: November 11, 2025
Myonuclear Domain-Associated and Central Nucleation-Dependent Spatial Restriction of Dystrophin Protein Expression.
Journal: Journal of cachexia, sarcopenia and muscle
Published: October 02, 2025
Design, validation, and functional impact of oligonucleotides for multigene silencing in Alzheimer's disease.
Journal: Molecular therapy. Nucleic acids
Published: September 26, 2025
Galectin-3 induces neurodevelopmental apical-basal polarity and regulates gyrification.
Journal: Science advances
Published: September 03, 2025
Biophysical and biological properties of splice-switching oligonucleotides and click conjugates containing LNA-phosphothiotriester linkages.
Journal: Nucleic acids research
Published: August 11, 2025
Systemic Inflammation Modulates Clearance and Drives Extra-Hepatic Distribution of Extracellular Vesicles.
Journal: Journal of extracellular vesicles
Published: August 07, 2025
Synthesis, Biological Activity, and Molecular Dynamics Simulations of LNA-Charge Neutral Linkages for Enhanced Splice-Switching Antisense Oligonucleotides.
Journal: Angewandte Chemie (International ed. in English)
Published: May 24, 2025
The Rare Therapies Launchpad: a pilot program for individualized medicines in the UK.
Journal: Nature medicine
Published: March 11, 2025
Last Updated: 09/26/2026