Search results (61)
« Back to PublicationsAccurately modeling RNase H-mediated antisense oligonucleotide efficacy.
Hill B. et al, (2026), Mol Ther Nucleic Acids, 37
Muscle MRI as an Imaging Biomarker of Muscle Damage in Patients With Spinal and Bulbar Muscular Atrophy.
Grunseich C. et al, (2026), Neurology, 107
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
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
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