Fen-Biao Gao, PhD
iPSC-Derived Motor Neuron Models of ALS with C9ORF72 Repeat Expansion
A repeat expansion in the C9ORF72 gene is the most common causal mutation in amyotrophic lateral sclerosis and frontotemporal dementia. However, no one knows exactly how this mutation causes disease. We will use Drosophila as a model system to investigate neurotoxicity of these repeat expansions. We hope these studies will contribute to the development of potential therapeutic interventions.
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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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