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The development of peptide- and oligonucleotide-based drugs to prevent the formation of abnormal tau in tauopathies.

Madia LozuponeVittorio DibelloRodolfo SardoneFabio CastellanaRoberta ZupoLuisa LampignanoIlaria BortoneRoberta StalloneMario AltamuraAntonello BellomoAntonio DanieleVincenzo SolfrizziFrancesco Panza
Published in: Expert opinion on drug discovery (2023)
ASOs are a promising therapeutic approach for tauopathies, particularly because ASOs may suppress the expression of harmful genes and are directly delivered to the brain, showing little systemic side effects. However, whether a generalized brain tau decrease will produce positive clinical effects remains unclear. A Phase II trial of BIIB080 is ongoing in mild AD. Neurotrophic and neurogenic peptide mimetic compounds have also shown potential as treatment options for AD and other tauopathies.
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