NEW PHARMACOLOGICAL TARGETS FOR CYSTIC FIBROSIS TREATMENT FROM EXTENSIVE PROTEOMIC PROFILING OF ?F508-CFTR EXPRESSING CELLS

摘要

Cystic fibrosis (CF) is a genetic disorder, caused by mutations in the CFTR gene [1]. Deletion of phenylalanine 508 (F508del) is the most frequent mutation. Dr. Pedemonte's group has selected molecules with a role in F508del-CFTR processing and degradation. After their silencing, an increase of CFTR at the cell membrane was measured [2], [3]. Using SWATH workflow [4], it is possible to analyze changes at proteome level. The first step is the creation of an ion library.