Ongoing research in the Neurobiology of Neurodegenerative Diseases Laboratory led by Wilfried Rossoll, Ph.D., is focused on understanding molecular disease mechanisms and developing novel therapeutic strategies for neurodegenerative diseases such as spinal muscular atrophy (SMA), amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD) and Alzheimer's disease (AD).
Dr. Rossoll and his team are involved in the following research projects:
- Dissecting molecular disease mechanisms in SMA. Spinal muscular atrophy (SMA) is the most common genetic cause of death for infants. Dr. Rossoll's research team is using advanced cellular and animal models to study the molecular functions of the disease protein SMN. The team is also focused on how reduced levels of this protein cause a deficiency in the assembly of RNA-protein complexes and the progressive degeneration of motor neurons in SMA.
- Identifying mechanisms of protein aggregation in neurodegenerative diseases. Research in Dr. Rossoll's lab is focused on aggregation-prone proteins such as TDP-43 and tau, which are critically involved in the pathogenesis of ALS, FTD and AD. Studies in the lab seek to gain a detailed understanding of the aggregate-associated proteome, mechanisms that promote the aggregation, and how to prevent the pathology from occurring and spreading across the central nervous system.
- Developing modifiers of neurodegenerative diseases as novel therapeutic targets. Dr. Rossoll's team has discovered potent modifiers of pathological TDP-43 aggregation, which is a hallmark of FTD, ALS and other neurodegenerative diseases. His team is utilizing cutting-edge techniques in the areas of biochemistry, molecular and cell biology, and fluorescence microscopy to identify ways to prevent the formation of pathological protein aggregates in various cell models including patient-derived cells and in vivo models.