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Wilfried O. Rossoll, Ph.D., and colleagues study molecular mechanisms and cellular pathways of neurodegeneration in diseases characterized either by reduced assembly of RNA and proteins, such as spinal muscular atrophy (SMA), or by increased protein aggregation, such as amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD) and Alzheimer's disease.
Members of Dr. Rossoll's Neurobiology of Neurodegenerative Diseases Laboratory use model systems such as cell culture, mouse models and stem cell-derived neurons from human patients to understand how dysfunction in RNA processing and protein homeostasis can cause neurodegenerative diseases. As researchers learn more about disease mechanisms, the focus is shifting to translational research to develop new therapeutic approaches for treatment and prevention.
The unmet need for effective therapies to treat the growing number of patients with neurodegenerative diseases is creating a mounting personal and financial burden on society. Among the most common hallmarks of these diseases are defects in RNA processing and the pathological aggregation of proteins. Research in Dr. Rossoll's lab is dedicated to providing a better understanding of these processes, which is an essential step in the development of effective novel therapeutic strategies for patients with neurodegenerative disorders.
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