Article

PP-2, a src-kinase inhibitor, is a potential corrector for F508del-CFTR in cystic fibrosis

2018-03-24

Abstract excerpt

Cystic fibrosis (CF) is an autosomal recessive disorder caused by mutations in the CF transmembrane conductance regulator (CFTR) gene. The most common mutation in CF, an in-frame deletion of phenylalanine 508, leads to a trafficking defect and endoplasmic reticulum retention of the protein where it becomes targeted for degradation. Successful clinical deployments of ivacaftor and ivacaftor/lumacaftor combination h...

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Literature Corpus work: 956356d2-8dc3-5be9-b92b-81f695e93653
DOI: 10.1101/288324

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