Davide Trotti, PhD & Ya-Ming Hou, PhD
Deciphering RAN translation and tRNA requirements in C9orf72-ALS/FTD tractable in vitro models
An hexa-nucleotide repeat expansion in the C9orf72 gene in amyotrophic lateral sclerosis and frontotemporal dementia leads to the production of neurotoxic peptides through a process called repeat-associated non-AUG (RAN) translation. The genesis of this process is under intense investigations. However, progress is in general hampered primarily by the lack of experimental tractable models. To overcome this gap, we propose to develop and validate in vitro cell-based and cell-free experimental models of RAN translation that afford quantitative as well as qualitative assays. Moreover, these tractable models will allow us to investigate distinct facets of RAN translation and to produce comprehensive data that will guide the advancement of potential novel treatments.
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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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