Lesley A. Kane

This study shows SARM1 causes calpain-dependent dendrite degeneration in mouse hippocampal neurons, revealing distinct degeneration pathways in axons and dendrites.

DNL343 reduces neurodegeneration in mouse models by inhibiting the integrated stress response and normalizing dysregulated biomarkers, with potential clinical relevance for vanishing white matter disease.

This paper demonstrates that systemically delivered OTV, a TfR-binding molecule, effectively transports ASO across the BBB, achieving widespread and sustained knockdown of target genes in the CNS of mice and non-human primates.