Joanna A. Korecka

This study uses a humanized mouse model to show that antisense oligonucleotides reduce seizure activity and mortality in DRPLA.

This paper demonstrates that antisense oligonucleotides targeting human ATN1 protect against DRPLA pathology in a fully humanized mouse model.

This study demonstrates that BH4 deficiency exacerbates PD-related stressors and that BH4 augmentation protects dopaminergic neurons in multiple models.