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

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 th...

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Autores principales:	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
Formato:	article
Lenguaje:	EN
Publicado:	Nature Portfolio 2016
Materias:	Science Q
Acceso en línea:	https://doaj.org/article/837ff0b26f1b42a594f1b539d90bcb7c
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spelling	oai:doaj.org-article:837ff0b26f1b42a594f1b539d90bcb7c2021-12-02T17:32:05ZRepositioning tolcapone as a potent inhibitor of transthyretin amyloidogenesis and associated cellular toxicity10.1038/ncomms107872041-1723https://doaj.org/article/837ff0b26f1b42a594f1b539d90bcb7c2016-02-01T00:00:00Zhttps://doi.org/10.1038/ncomms10787https://doaj.org/toc/2041-1723Misfolding 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.Ricardo Sant'AnnaPablo GallegoLei Z. RobinsonAlda Pereira-HenriquesNelson FerreiraFrancisca PinheiroSebastian EsperanteIrantzu PallaresOscar HuertasMaria Rosário AlmeidaNatàlia ReixachRaul InsaAdrian Velazquez-CampoyDavid ReverterNúria ReigSalvador VenturaNature PortfolioarticleScienceQENNature Communications, Vol 7, Iss 1, Pp 1-13 (2016)
institution	DOAJ
collection	DOAJ
language	EN
topic	Science Q
spellingShingle	Science Q 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 Repositioning tolcapone as a potent inhibitor of transthyretin amyloidogenesis and associated cellular toxicity
description	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.
format	article
author	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
author_facet	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
author_sort	Ricardo Sant'Anna
title	Repositioning tolcapone as a potent inhibitor of transthyretin amyloidogenesis and associated cellular toxicity
title_short	Repositioning tolcapone as a potent inhibitor of transthyretin amyloidogenesis and associated cellular toxicity
title_full	Repositioning tolcapone as a potent inhibitor of transthyretin amyloidogenesis and associated cellular toxicity
title_fullStr	Repositioning tolcapone as a potent inhibitor of transthyretin amyloidogenesis and associated cellular toxicity
title_full_unstemmed	Repositioning tolcapone as a potent inhibitor of transthyretin amyloidogenesis and associated cellular toxicity
title_sort	repositioning tolcapone as a potent inhibitor of transthyretin amyloidogenesis and associated cellular toxicity
publisher	Nature Portfolio
publishDate	2016
url	https://doaj.org/article/837ff0b26f1b42a594f1b539d90bcb7c
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Repositioning tolcapone as a potent inhibitor of transthyretin amyloidogenesis and associated cellular toxicity

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