Leonard Petrucelli, PhD
The role of epigenetic changes and downstream pathological events in c9FTD/ALS
Amyotrophic lateral sclerosis (ALS) is an incurable disease of nerve cells that control muscles, and some patients with ALS have mental and behavioral abnormalities similar to frontotemporal dementia (FTD). We aim to discover the cellular disease processes initiated by mutations in the C9ORF72 gene in order to improve the diagnosis of and prognosis for patients suffering from disorders collectively referred to as c9FTD/ALS. By better understanding cellular disease processes known as epigenetic changes, we hope to identify new biomarkers and treatment therapies.
Note: Funding for this project is made possible by Ann Arbor Active Against ALS (A2A3)
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Gladstone Institutes, UCSF
Two recently discovered genes that have been associated with both familial and sporadic forms of ALS encode the related proteins TDP43 and FUS cause neuron death in ALS.
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