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Search results (124)

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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

Evidence that corticofugal propagation of ALS pathology is not mediated by prion-like mechanism.

Scekic-Zahirovic J. et al, (2020), Prog Neurobiol

Cooperation and Interplay between EGFR Signalling and Extracellular Vesicle Biogenesis in Cancer.

Zanetti-Domingues LC. et al, (2020), Cells, 9

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

Mechanisms of Action of EGFR Tyrosine Kinase Receptor Incorporated in Extracellular Vesicles.

Zanetti-Domingues LC. et al, (2020), Cells, 9

A Single Amino Acid Residue Regulates PTEN-Binding and Stability of the Spinal Muscular Atrophy Protein SMN.

Rademacher S. et al, (2020), Cells, 9

Human muscle signaling responses to 3-day head-out dry immersion

Vilchinskaya NA. et al, (2020), The Journal of Musculoskeletal and Neuronal Interactions, 15

Advances in oligonucleotide drug delivery.

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

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, 144

Relative contribution of ancient woodland indicator and non-indicator species to herb layer distinctiveness in ancient semi-natural, ancient replanted, and recent woodland

Swallow KA. et al, (2020), Applied Vegetation Science, 23, 471 - 481

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

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

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

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

Absence of subcerebral projection neurons is beneficial in a mouse model of ALS.

Burg T. et al, (2020), Ann Neurol

Combined Treatment with Peptide-Conjugated Phosphorodiamidate Morpholino Oligomer-PPMO and AAV-U7 Rescues the Severe DMD Phenotype in Mice

Forand A. et al, (2020), Molecular Therapy - Methods and Clinical Development, 17, 695 - 708

Linkers

VARELA MA., (2020)

Linkers

VARELA MA., (2020)

Gene therapies for polyglutamine diseases

Matos C. et al, (2020), Polyglutamine Disorders

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

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

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