Recent Advance in Basic Science
Valeria Raia (1), Luigi Maiuri (2,3)
1Dipartimento di Pediatria, Centro Regionale Fibrosi Cistica, Università di Napoli "Federico II"2European Institute for Research in Cystic Fibrosis, Istituto Scientifico San Raffaele di Milano3Dipartimento di Scienze Mediche e Chirurgiche, Università di Foggia
We focus on the manipulation of proteostasis and autophagy as a new option of CFTR repairing therapy to rescue functional F508delCFTR in CF. We highlight how targeting the intracellular environment surrounding the misfolded mutant CFTR instead of protein itself could constitute an attractive therapeutic option to sensitize F508delCFTR patients to the beneficial action of CFTR potentiators on lung inflammation.