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Development of novel methodologies for the identification of CFTR-targeted drugs: a multi
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Sala D, Rusnati M, Orro A, Trombetti G, Cichero E, Urbinati C, Di Somma M, Millo E, Galietta LJV , Milanesi L, Fossa P, D’Ursi P
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Univ. of Brescia, Univ. of Genoa
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ITB-CNR, Segrate MI; DMMT, Univ. of Brescia; Pharmacy, Exp Med, CEBR, Univ. of Genoa; Ist. G. Gaslini, Genoa, Italy
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info@postdocjournal.com
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Background. Cystic fibrosis (CF) is due to dysfunctions of CFTR whose most common mutation, F508Δ, localized in NBD1 domain, causes an abnormal conformation of F508Δ-CFTR that is withhold in the endoplasmic reticulum. The small amount of receptor that reaches the plasma membrane exhibits low activity (gating defect).
Hypothesis & objectives. Current CF therapies are aimed at symptoms alleviation, calling for new drugs to rescue F508Δ-CFTR trafficking (correctors) or gating (potentiators), to the identification of which this study has sought to make an original contribution.
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