Miquéias Lopes-Pacheco

@mlopes0811.bsky.social

Brother of a CF warrior (in memoriam) | MS, PhD | Drug Development and Precision Medicine | Respiratory Disease Researcher | Cat lover | Traveler | 40 years.

(1) EN: Elexacaftor/tezacaftor/ivacaftor (ETI) and vanzacaftor/tezacaftor/deutivacaftor (VTD) are the two most effective CFTR modulator combinations to date. Both pair two correctors with complementary mechanisms and one potentiator, producing synergistic rescue of F508del-CFTR folding and function.

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(1) EN: Ivacaftor is a potentiator that binds directly to CFTR protein at cell surface, increasing channel open probability and thereby enhancing chloride transport. It was identified by high-throughput screening for the G551D variant and later evaluated in other gating and conductance variants.

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(1) EN: October is Primary Ciliary Dyskinesia (PCD) Awareness Month. PCD is a rare genetic condition in which cilia don’t beat properly, often leading to chronic lung infections, bronchiectasis, and, in some cases, situs inversus. Because testing is limited, diagnosis can take a long time.

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EN: I am thrilled to share that Scholarly Index has recognized me among the Top 1000 Scholars in two research topics: 1) Cystic Fibrosis Research Advances — World Rank #56 (2021–2025) and #756 (lifetime) 🧬 2) Neonatal Respiratory Health Research — World Rank #362 (2021–2025) 🚼

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(1) EN: In 1989, three Science papers identified the Cystic Fibrosis (CF) gene using complementary approaches: 1) chromosome walking and jumping mapped the locus to chromosome 7; 2) cDNA cloning revealed a ~6,500-nucleotide transcript coding a protein with 2 transmembrane and 2 ATP-binding domains;

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