November 2024
- Our latest preprint is now out ! We identified the Pml gene as a key genetic interactor in ALS (Amyotrophic Lateral Sclerosis), protecting mice from disease onset by clearing misfolded proteins in motoneurons. We also found that PML-targeting agents hold promise as a potential therapy for ALS, a devastating neurodegenerative disease with no effective treatment. “Pharmacological intervention aimed at augmenting Pml expression, achieved through interferon alpha (IFN) or poly(I:C) treatment, effectively eliminates NEK1t and its aggregates from motoneurons in vivo. This intervention dramatically improves ALS- associated symptoms and muscle strength, and extends survival by nearly 6 months, in a Pml-dependent manner. To our knowledge, this is the most dramatic benefit ever reported in treatment trials initiated presymptomatically in ALS mouse models. “
