NeurosciencebioRxivPreprint
Type I PRMT inhibitor MS023 promotes SMN2 exon 7 inclusion and synergizes with nusinersen to rescue the phenotype of SMA mice
Anna J. Kordala, Nina Ahlskog, Muhammad Hanifi…·Carlo Rinaldi
1 citation
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John Radcliffe HospitalORCID 0000-0002-5640-7569Unclaimed ORCID profile · follow to get notified of new preprints
3 papers
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Anna J. Kordala, Nina Ahlskog, Muhammad Hanifi…·Carlo Rinaldi
Suzan M. Hammond, Frank Abendroth, Larissa Goli…·Carl I. Webster
This study demonstrates that systemic delivery of ASO conjugated to a brain-targeting antibody improves SMN2 splicing and survival in SMA mouse models.
Katharina E. Meijboom, Viola Volpato, Jimena Monzón‐Sandoval…·Mélissa Bowerman
This study identifies novel drug treatments for spinal muscular atrophy by integrating multi-omics data and drug perturbation profiles, showing harmine improves disease phenotypes in models.