FFC#4/2014

The molecular structure and the folding of the whole Cystic Fibrosis Transmembrane Conductance Regulator (CFTR): correctors sites

FFC#4/2014

The molecular structure and the folding of the whole Cystic Fibrosis Transmembrane Conductance Regulator (CFTR): correctors sites

PRINCIPAL INVESTIGATOR

Oscar Moran (Istituto di Biofisica, Consiglio Nazionale delle Ricerche – CNR, Genova)

RESEARCHERS

5

CATEGORY

AREA 1 Therapies to correct the underlying defect

DURATION

2 years

GOAL

€ 45.000

RESULTS

Remarkable characteristics in the conformation of normal CFTR and full length mutant F508del mutant were unravelled, in contrast with the minor differences observed for the isolated F508del containing domain. New data about the mutant may be interpreted in terms of an incomplete post-translational assembly of the protein domains. It follows that the use of wild type molecular models to find better correctors must be reconsidered.

OTHER RESULTS

FFC #3/2024

Two molecules are effective in activating Heat Shock Proteins and enhancing the action of CFTR correctors with the F508del mutation in vitro.

FFC#5/2024

Some peptide nucleic acids (PNAs) re-sensitise Pseudomonas aeruginosa to the antibiotic meropenem in vitro and reduce its virulence.

FFC#1/2023

Tezacaftor, one of the components of Kaftrio, induces an accumulation of dihydroceramides both in vitro and in vivo in animal models