The only approved potentiator drug for gating mutations, VX-770, provides significant, albeit partial, restoration of the channel function for the most common gating mutant, G551D-CFTR, while partially inhibits the long-term lung function decay. Several rare gating mutations are marginally responsive to the VX-770 activation but, based on published and preliminary data, we posit that the […]

Last generation 4,6,4′- trimethylangelicin (TMA) – derivatives with anti-inflammatory properties  update April 2023 Correction of the defective CFTR protein is the priority goal in the treatment of the lung pathology of patients affected by cystic fibrosis. The possibility that mutated CFTR correctors and potentiators currently in clinics are sufficient to intervene on the chronic inflammatory […]

mitochondria as central hub of pulmonary inflammation in cystic fibrosis The ideal drug or treatment for cystic fibrosis (CF) should be one that rescues and potentiates the residual functionality of defective CF Transmembrane conductance Regulator (CFTR) channel, but also generates favourable conditions to interrupt the vicious circle that sustains lung hyperinflammation generated by chronic bacterial infection and […]

Peptide-nucleic acids for CFTR correction MicroRNAs as key post-transcriptional regulators of gene expression. MicroRNAs (miRNAs) are a family of small (19 to 25 nucleotides in length) noncoding RNAs that regulate gene expression by sequence-selective targeting of mRNAs, leading to a translational repression or mRNA degradation, depending on the degree of complementarity between miRNAs and the […]

when old drugs learn new tricks: betasitosterol (BSS) from traditional medicine to anti-inflammatory molecular therapy: a safe pharmaceutical repurposing update April 2023 By serendipity, looking at the amazingly positive clinical response in a native-Morrocan child affected by a chronic inflammatory lung disease when she was assuming per os the “black seeds from North-Africa deserts”, a traditional […]