Kathleen M. Schoch

The paper demonstrates that antisense oligonucleotides (ASOs) can upregulate gene expression by stabilizing mRNA through targeting destabilizing elements like PREs, with therapeutic implications for haploinsufficiency disorders.

The paper shows miR-146a regulates multiple aspects of motor neuron degeneration in ALS, with genetic deletion extending survival and causing age-related paralysis and inflammation.

This study develops a lipid-siRNA conjugate that uses perivascular transport to achieve long-lasting gene knockdown in the CNS without toxicity.

This study evaluates the efficacy of commercially available antisense oligonucleotides (ASOs) targeting mouse and human tau in vivo, showing reduced tau levels and pathology in mouse models.

This study identifies TSC1 loss-of-function as a risk factor for tauopathies by promoting tau acetylation and impairing autophagy-mediated tau clearance.