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RNase-H-mediated silencing in the CNS proves predictably nontrivial.

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Moazami MP. and Wood MJA., (2022), Med (N Y), 3, 733 - 734

Exon skipping induces uniform dystrophin rescue with dose-dependent restoration of serum miRNA biomarkers and muscle biophysical properties

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Chwalenia K. et al, (2022), Molecular Therapy - Nucleic Acids, 29, 955 - 968

Dysregulation of Tweak and Fn14 in skeletal muscle of spinal muscular atrophy mice.

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Meijboom KE. et al, (2022), Skelet Muscle, 12

AR cooperates with SMAD4 to maintain skeletal muscle homeostasis.

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Forouhan M. et al, (2022), Acta Neuropathol, 143, 713 - 731

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

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

Control of backbone chemistry and chirality boost oligonucleotide splice switching activity.

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Kandasamy P. et al, (2022), Nucleic Acids Res

Enhancing the Therapeutic Potential of Extracellular Vesicles Using Peptide Technology


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

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

Dystrophin involvement in peripheral circadian SRF signalling.

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Betts CA. et al, (2021), Life Sci Alliance, 4

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