AudioAbstracts

Cell Death and Differentiation: A Novel Treatment of Cystic Fibrosis Acting On-Target

July 31, 20163 min
A novel treatment of cystic fibrosis acting on-target: cysteamine plus epigallocatechin gallate for the autophagy-dependent rescue of class II-mutated CFTR Tosco A1, De Gregorio F1, Esposito S2, De Stefano D2, Sana I2, Ferrari E2, Sepe A1, Salvadori L1, Buonpensiero P1, Di Pasqua A1, Grassia R3, Leone CA3,Guido S4, De Rosa G5, Lusa S6, Bona G7, Stoll G8,9,10,11, Maiuri MC9, Mehta A12, Kroemer G9,13,14,15,16,17, Maiuri L2,7, Raia V1. Cell Death Differ. 2016 Aug;23(8):1380-93. doi: 10.1038/cdd.2016.22. Epub 2016 Apr 1. Interpretation: Cystic fibrosis, the most common lethal recessive disease in Caucasians, affects approximately 70,000 patients worldwide and results from mutations in the cystic fibrosis transmembrane conductance regulator (CFTR). For the vast majority of cystic fibrosis patients bearing the most common Phe508del-CFTR mutation, an FDA-approved combined treatment with the corrector Lumacaftor (that promotes ER to plasma membrane traffic) and Ivacaftor is only marginally ef

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