Repositioning tolcapone as a potent inhibitor of transthyretin amyloidogenesis and associated cellular toxicity

Autor: Ricardo Sant'Anna, Pablo Gallego, Lei Z. Robinson, Alda Pereira-Henriques, Nelson Ferreira, Francisca Pinheiro, Sebastian Esperante, Irantzu Pallares, Oscar Huertas, Maria Rosário Almeida, Natàlia Reixach, Raul Insa, Adrian Velazquez-Campoy, David Reverter, Núria Reig, Salvador Ventura
Jazyk: angličtina
Rok vydání: 2016
Předmět:
Zdroj: Nature Communications, Vol 7, Iss 1, Pp 1-13 (2016)
Druh dokumentu: article
ISSN: 2041-1723
DOI: 10.1038/ncomms10787
Popis: Misfolding of transthyretin can cause amyloid aggregation disorders that can be treated by stabilizing the tetrameric form with tafamidis. Here the authors show that tolcapone, a drug already FDA-approved for Parkinson disease, has strong transthyretin stabilizing function and might be a superior therapeutic option for CNS amyloidosis as it can cross the blood brain barrier.
Databáze: Directory of Open Access Journals