Identification and validation of novel molecules obtained by integrated computational and experimental approaches for the readthrough of PTCs in CF cells

Research output: Contribution to conferenceOther

Abstract

Cystic Fibrosis patients with nonsense-mutation in h-CFTR gene generally make virtually no CFTR protein and thus often have a more severe form of CF. Ataluren (PTC124) was suggested to induce read-through of premature but not normal termination codons. Despite the promising results there is not a general consensus on the mechanism of its action (protein stabilization or codon read-through) and its efficacy, the identification of new PTC124 analogues and the study of the mechanism of action may led to a new strategy for the development of a pharmacologic approach to the cure of CF.
Original languageEnglish
Number of pages1
Publication statusPublished - 2014

Fingerprint Dive into the research topics of 'Identification and validation of novel molecules obtained by integrated computational and experimental approaches for the readthrough of PTCs in CF cells'. Together they form a unique fingerprint.

Cite this