Mario Plaas Research Group

The Plaas Group (University of Tartu) – Translational research for Wolfram syndrome 1

Wolfram syndrome 1 (WS1) is a rare genetic disorder that often begins with childhood-onset diabetes and can progress to vision and hearing loss and neurodegeneration. There is still no effective disease-modifying therapy, so new treatment strategies are urgently needed.

Professor Mario Plaas is a pioneer in WS1 preclinical research, internationally recognized for creating and validating foundational WS animal models and for leading in vivo drug testing to identify therapies that can slow disease progression. His team developed landmark Wfs1 knockout mouse and rat models that reproduce key WS1 features across the lifespan, enabling rigorous, long-term evaluation of candidate treatments.

The group studies how endoplasmic reticulum (ER) stress, disrupted cellular cargo/trafficking, neuroinflammation, and impaired proteostasis (including autophagy) contribute to WS1—potentially alongside an imbalance between excitatory and inhibitory neurotransmission. Building on these mechanisms, the Plaas group tests combination regimens, including incretin-based therapies together with approaches targeting GABAergic vulnerability and cellular stress/autophagy pathways, aiming to slow or halt both metabolic decline and neurodegeneration.

Recognition: Plaas has received major national honors for his translational and preclinical research, including the Estonian National Science Award in Medicine (2021) and the Estonian Academy of Sciences Young Scientist Award (2021).

Collaborations