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Philip C. Wong, PhD

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Philip C. Wong, PhD

Johns Hopkins University

Validation of a therapeutic strategy to repress TDP-43 cryptic exons for ALS

Recent studies indicate that a critical role to repress cryptic exons of an RNA binding protein, termed TDP-43 linked to ALS, is compromised in brains of patients with ALS. In order to fix this potential problem in ALS, we have identified a potential strategy to restore this ability to repress cryptic exons in cells missing TDP-43. We plan to validate this therapeutic strategy in mice missing this role of TDP-43 in their motor neurons and positive outcomes from this proof-of-principal study will offer potential therapeutic strategies to attenuate this devastating disease of the elderly.

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Steven Finkbeiner

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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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