Repositioning tolcapone as a potent inhibitor of transthyretin amyloidogenesis and associated cellular toxicity
oleh: 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
| Format: | Article |
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| Diterbitkan: | Nature Portfolio 2016-02-01 |
Deskripsi
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.