Nina Ahlskog

This study develops a click chemistry-based strategy for siRNA conjugation to enhance extrahepatic RNAi, finding that bis-fatty-acid conjugates improve activity in heart and muscle while showing modality-specific limitations.

The paper tests uORF-targeting ASOs for RNASEH1 upregulation but finds no reproducible activation, instead observing downregulation in some cases.

This study shows that Fn14-depletion improves survival and muscle pathology in SOD1G93A ALS mice, but only when combined with exercise.

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.

This study demonstrates that systemic delivery of ASO conjugated to a brain-targeting antibody improves SMN2 splicing and survival in SMA mouse models.

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.