Restoration of CFTR function in patients with cystic fibrosis carrying the F508del-CFTR mutation.

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Published in Autophagy on January 01, 2014

Authors

Daniela De Stefano1, Valeria R Villella, Speranza Esposito, Antonella Tosco, Angela Sepe, Fabiola De Gregorio, Laura Salvadori, Rosa Grassia, Carlo A Leone, Giuseppe De Rosa, Maria C Maiuri, Massimo Pettoello-Mantovani, Stefano Guido, Anna Bossi, Anna Zolin, Andrea Venerando, Lorenzo A Pinna, Anil Mehta, Gianni Bona, Guido Kroemer, Luigi Maiuri, Valeria Raia

Author Affiliations

1: a European Institute for Research in Cystic Fibrosis; Division of Genetics and Cell Biology; San Raffaele Scientific Institute ; Milan , Italy.

Associated clinical trials:

Dose-escalation Study of Oral CX-4945 | NCT00891280

Study of CX-4945 in Patients With Relapsed or Refractory Multiple Myeloma | NCT01199718

Efficacy and Safety Study of EGCG/Tocotrienol in 18 Patients With Splicing-mutation-mediated Cystic Fibrosis (CF) | NCT00889434

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