Cookies on this website

We use cookies to ensure that we give you the best experience on our website. If you click 'Continue' we'll assume that you are happy to receive all cookies and you won't see this message again. Click 'Find out more' for information on how to change your cookie settings.

Publications

Gene therapy with AR isoform 2 rescues spinal and bulbar muscular atrophy phenotype by modulating AR transcriptional activity.

Lim WF. et al, (2021), Sci Adv, 7

Combining multiomics and drug perturbation profiles to identify muscle-specific treatments for spinal muscular atrophy.

Meijboom KE. et al, (2021), JCI Insight, 6

Mesyl Phosphoramidate Oligonucleotides as Potential Splice Switching Agents: Impact of Backbone Structure on Activity and Intracellular Localization

HAMMOND SM. et al, (2021), Nucleic Acid Therapeutics

Extracellular vesicles as a next-generation drug delivery platform

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

Emerging Oligonucleotide Therapeutics for Rare Neuromuscular Diseases.

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

Mesyl Phosphoramidate Oligonucleotides as Potential Splice-Switching Agents: Impact of Backbone Structure on Activity and Intracellular Localization.

Hammond SM. et al, (2021), Nucleic Acid Ther

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

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

Immortalized Canine Dystrophic Myoblast Cell Lines for Development of Peptide-Conjugated Splice-Switching Oligonucleotides.

Tone Y. et al, (2021), Nucleic Acid Ther

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

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

Molecular and electrophysiological features of spinocerebellar ataxia type seven in induced pluripotent stem cells.

Burman RJ. et al, (2021), PLoS One, 16

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

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

Erratum: Context Dependent Effects of Chimeric Peptide Morpholino Conjugates Contribute to Dystrophin Exon-skipping Efficiency (Context Dependent Effects of Chimeric Peptide Morpholino Conjugates Contribute to Dystrophin Exon-skipping Efficiency (2013) 2 (1–10), (S2162253116301834), (10.1038/mtna.2013.51))

Yin HF. et al, (2020), Molecular Therapy - Nucleic Acids, 22

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

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

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

Engineered extracellular vesicle decoy receptor-mediated modulation of the IL6 trans-signalling pathway in muscle.

Conceição M. et al, (2020), Biomaterials, 266

Advances in oligonucleotide drug delivery.

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

Mutation-independent proteomic signatures of pathological progression in murine models of Duchenne muscular dystrophy.

van Westering TLE. et al, (2020), Mol Cell Proteomics

An ALS-linked mutation in TDP-43 disrupts normal protein interactions in the motor neuron response to oxidative stress.

Feneberg E. et al, (2020), Neurobiol Dis

Efficient Doxorubicin Loading to Isolated Dexosomes of Immature JAWSII Cells: Formulated and Characterized as the Bionanomaterial.

Mutlu EC. et al, (2020), Materials (Basel), 13

Novel EGFP reporter cell and mouse models for sensitive imaging and quantification of exon skipping

Hara Y. et al, (2020), Scientific Reports

Load More

1
2
3
4
5
6
7
...
»

Include forthcoming?