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Muscle MRI as an Imaging Biomarker of Muscle Damage in Patients With Spinal and Bulbar Muscular Atrophy.
Journal article
Grunseich C. et al, (2026), Neurology, 107
Timing matters: exon skipping therapy is most effective when initiated early in a mouse model of Duchenne muscular dystrophy
Journal article
Stenler S. et al, (2026), Molecular Therapy Nucleic Acids, 103017 - 103017
Click Chemistry-Based Strategy for Modular Ligand Attachment to siRNAs: Toward Extrahepatic RNAi
Preprint
Rädler JA. et al, (2026)
Systemic Inflammation Modulates Clearance and Drives Extra-Hepatic Distribution of Extracellular Vesicles.
Journal article
Pavlova S. et al, (2026), J Extracell Vesicles, 15
Myonuclear Domain-Associated and Central Nucleation-Dependent Spatial Restriction of Dystrophin Protein Expression.
Journal article
Chwalenia K. et al, (2026), J Cachexia Sarcopenia Muscle, 17
Design, validation, and functional impact of oligonucleotides for multigene silencing in Alzheimer's disease.
Journal article
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.
Journal article
González-Martínez I. et al, (2026), Am J Hum Genet, 113, 529 - 547
Targeted BDNF upregulation via upstream open reading frame disruption.
Journal article
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
Preprint
Stenler S. et al, (2025)
The CTDP1 Founder Variant in CCFDN: Insights into Pathogenesis, Phenotypic Spectrum and Therapeutic Approaches.
Journal article
Sabau IM. et al, (2025), Int J Mol Sci, 27
Exon skipping peptide-conjugated morpholinos downregulate dynamin 2 to rescue centronuclear myopathy.
Journal article
Moschovaki-Filippidou F. et al, (2025), Brain, 148, 4495 - 4507
Progress and prospects in antisense oligonucleotide-mediated exon skipping therapies for Duchenne muscular dystrophy.
Journal article
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.
Journal article
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.
Journal article
Kennett A. et al, (2025), Angew Chem Int Ed Engl, 64
Accurately modelling RNase H-mediated antisense oligonucleotide efficacy
Preprint
Hill B. et al, (2025)
Thrombospondin Encapsulated Dense Particles accelerate wound healing and downregulate fibrotic phenotypes
Preprint
Staton CC. et al, (2025)
AAV microdystrophin gene replacement therapy for Duchenne muscular dystrophy: progress and prospects.
Journal article
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.
Journal article
Moreno N. et al, (2025), Mol Ther Nucleic Acids, 36
Galectin-3 induces neurodevelopmental apical-basal polarity and regulates gyrification.
Journal article
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
Preprint
Chwalenia K. et al, (2025)
