Anna M. L. Coenen-Stass

This study shows that exon skipping therapy is more effective when started early in adult mdx mice than in aged mice, leading to better dystrophin restoration and transcriptomic changes.

This study uses CRISPR to excise exon 23 in dKO mice, achieving partial dystrophin restoration but with limited efficacy and challenges in DNA repair.

This study shows that PPMO-mediated exon skipping in mdx mice restores uniform dystrophin expression and dose-dependently improves serum microRNA biomarkers and muscle biophysical properties.

SLIDE-VIP identifies synthetic lethal gene pairs in DNA damage repair, chromatin remodeling, and cell cycle using multi-omics data and eight statistical tests, reducing candidate pairs by 250-fold.

The paper identifies miR-181 as a prognostic biomarker in ALS, showing it predicts higher mortality risk and improves prognosis prediction when combined with NfL.