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Extracellular vesicles as a next-generation drug delivery platform

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WOOD M. et al, (2021), Nature Nanotechnology

Emerging Oligonucleotide Therapeutics for Rare Neuromuscular Diseases.

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Aoki Y. and Wood MJA., (2021), J Neuromuscul Dis

Targeting the 5' untranslated region of SMN2 as a therapeutic strategy for spinal muscular atrophy.

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Winkelsas AM. et al, (2021), Mol Ther Nucleic Acids, 23, 731 - 742

Molecular correction of Duchenne muscular dystrophy by splice modulation and gene editing.

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Hanson B. et al, (2021), RNA Biol, 1 - 15

Mesyl phosphoramidate backbone modified antisense oligonucleotides targeting miR-21 with enhanced in vivo therapeutic potency.

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Patutina OA. et al, (2020), Proc Natl Acad Sci U S A, 117, 32370 - 32379

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

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

Mutation-independent Proteomic Signatures of Pathological Progression in Murine Models of Duchenne Muscular Dystrophy.

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van Westering TLE. et al, (2020), Mol Cell Proteomics, 19, 2047 - 2068

Advances in oligonucleotide drug delivery.

Journal article

Roberts TC. et al, (2020), Nat Rev Drug Discov, 19, 673 - 694

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