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Search results (60)

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Muscle MRI as an Imaging Biomarker of Muscle Damage in Patients With Spinal and Bulbar Muscular Atrophy.

Grunseich C. et al, (2026), Neurology, 107

Accurately modelling RNase H-mediated antisense oligonucleotide efficacy

Hill B. et al, (2025)

Tissue-specific modulation of CRISPR activity by miRNA-sensing guide RNAs.

Garcia-Guerra A. et al, (2025), Nucleic Acids Res, 53

Tissue-specific modulation of CRISPR activity by miRNA-sensing guide RNAs

Garcia-Guerra A. et al, (2024)

PRMT inhibitor promotes SMN2 exon 7 inclusion and synergizes with nusinersen to rescue SMA mice.

Kordala AJ. et al, (2023), EMBO Mol Med, 15

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

LSD1/PRMT6-targeting gene therapy to attenuate androgen receptor toxic gain-of-function ameliorates spinobulbar muscular atrophy phenotypes in flies and mice.

Prakasam R. et al, (2023), Nat Commun, 14

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

Kandasamy P. et al, (2022), Nucleic Acids Res, 50, 5443 - 5466

AR cooperates with SMAD4 to maintain skeletal muscle homeostasis.

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

MicroRNA-298 reduces levels of human amyloid-β precursor protein (APP), β-site APP-converting enzyme 1 (BACE1) and specific tau protein moieties.

Chopra N. et al, (2021), Mol Psychiatry, 26, 5636 - 5657

Dystrophin involvement in peripheral circadian SRF signalling.

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

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

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

Variants in ATP6V0A1 cause progressive myoclonus epilepsy and developmental and epileptic encephalopathy.

Bott LC. et al, (2021), Brain Commun, 3

Plasma pNfH levels differentiate SBMA from ALS.

Lombardi V. et al, (2020), J Neurol Neurosurg Psychiatry, 91, 215 - 217

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