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Search results (47)

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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

Enhanced muscle uptake of chemically optimized miR-23b antisense oligonucleotides as lead compounds for myotonic dystrophy type 1.

González-Martínez I. et al, (2026), Am J Hum Genet

Enhanced muscle uptake of chemically optimized miR-23b antisense oligonucleotides as lead compounds for Myotonic Dystrophy type 1

González-Martínez I. et al, (2026)

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

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

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

Dysregulated MEG3 in Myotonic Dystrophy 1: nuclear retention, pathological role, and therapeutic correction by antisense conjugates

Seoane-Miraz D. et al, (2025)

TDP-43 regulates LC3ylation in neural tissue through ATG4B cryptic splicing inhibition.

Torres P. et al, (2024), Acta Neuropathol, 148

Peptide-conjugated antimiRs improve myotonic dystrophy type 1 phenotypes by promoting endogenous MBNL1 expression.

González-Martínez I. et al, (2023), Mol Ther Nucleic Acids, 34

A modular RNA delivery system comprising spherical nucleic acids built on endosome-escaping polymeric nanoparticles.

Garcia-Guerra A. et al, (2023), Nanoscale Adv, 5, 2941 - 2949

Application of Antisense Conjugates for the Treatment of Myotonic Dystrophy Type 1.

Stoodley J. et al, (2023), Int J Mol Sci, 24

Peptide-Conjugated PMOs for the Treatment of Myotonic Dystrophy.

Stoodley J. et al, (2023), Methods Mol Biol, 2587, 209 - 237

CELL-PENETRATING PEPTIDE CONJUGATES AND METHODS OF THEIR USE

VARELA MA., (2022)

Proof of concept of peptide-linked blockmiR-induced MBNL functional rescue in myotonic dystrophy type 1 mouse model.

Overby SJ. et al, (2022), Mol Ther Nucleic Acids, 27, 1146 - 1155

Preclinical characterization of antagomiR-218 as a potential treatment for myotonic dystrophy.

Cerro-Herreros E. et al, (2021), Mol Ther Nucleic Acids, 26, 174 - 191

Application of CRISPR-Cas9-Mediated Genome Editing for the Treatment of Myotonic Dystrophy Type 1.

Marsh S. et al, (2020), Mol Ther, 28, 2527 - 2539

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