(1) EN: Theratyping and theranostics are complementary tools that can bridge the gap between genotype and effective therapy. Theratyping classifies CFTR variants by drug responsiveness using cell lines, informing regulatory decisions.
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: Precision medicine is essential for cystic fibrosis (CF) because CFTR gene variants are highly heterogeneous. More than 2,100 variants exist, each producing distinct molecular defects, and they have traditionally been grouped into six functional classes.
(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.
(1) EN: Elexacaftor and Vanzacaftor are dual-function CFTR modulators that act as both correctors and weak co-potentiators. They bind to a unique pocket formed by TM2, TM10, TM11, and the lasso motif, distinct from the binding sites of Tezacaftor and Ivacaftor.
(1) EN: Lumacaftor and Tezacaftor are CFTR correctors with similar chemical structures that bind the same pocket in TMD1. By stabilizing F508del-CFTR folding, they promote the protein's exit from the endoplasmic reticulum and its trafficking to the cell surface.
(1) EN: World Mental Health Day 2026 (October 10) calls for "Lived experiences heard: real voices, real change." Mental and respiratory health are closely connected. People living with severe mental illness face significantly higher risks of COPD and asthma.
(1) EN: Yesterday (Oct 9) marked Lung Transplant Awareness Day 2026, honoring every step of the journey, from the waitlist to life after transplant. It brings together patients, caregivers, donors, and healthcare professionals who make this life-saving procedure possible.
(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.
EN: 🏆 I'm honored to share that, for the sixth consecutive year, I have been recognized among the Top 2% Cited Scientists worldwide, based on the list compiled by Stanford University using Scopus citation data. 👨🏻🔬🔬📊💊🫁 👉🏻 doi.org/10.17632/btc...
(1) EN: CFTR modulators have transformed care for people with Cystic Fibrosis (CF). Using high-throughput screening, researchers identified small-molecule correctors and potentiators that target specific defects in CF-causing variants.
(1) EN: Precision medicine is transforming respiratory care by making it more personalized, moving beyond broad disease labels to understand patients through biomarkers, endotypes, and treatable traits.
(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.
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) 🚼
(1) EN: Coffee may support your lung health more than you might expect. A dose-response study found that caffeine in coffee acts as a mild bronchodilator, improving FEV1 in people with asthma, though it is only about 40% as potent as theophylline.
(1) EN: Coffee breaks are more than downtime; they are welcoming spaces for scientific exchange. In the lab, isolation can breed stagnation; over coffee, failures, early-stage ideas, and doubts become shared puzzles to explore together.
(1) EN: October is Healthy Lung Month 2026. Our lungs work nonstop… So give them the care they deserve! Quit smoking or vaping, keep your home well-ventilated, stay active, and avoid secondhand smoke.
(1) EN: Every breath is a gift we often take for granted… until breathing becomes difficult! Today, we celebrate World Oxygen Day (O–2), the unseen element that sustains every cell in our bodies and every heartbeat.
(1) EN: The definitions of acute respiratory distress syndrome (ARDS) have evolved significantly since 1967, when Ashbaugh first described the syndrome clinically. In 1994, experts established a standard diagnostic approach based on blood oxygen levels.
#tbt to when I received the fantastic news that I was selected as a winner of the 2025 International Journal of Molecular Sciences (IJMS) Young Investigator Award! 👨🏻🔬🏆 #IJMS #YoungInvestigator #MolecularMedicine #BiomedicalResearch @mdpiopenaccess.bsky.social www.mdpi.com/journal/ijms...
(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;
(1) EN: A recent study highlights that, despite decades of progress in reducing chronic respiratory disease (CRD) mortality, the burden remains substantial, with increasing concentration among older people and in certain regions.
(1) EN: Respiratory diseases are responsible for millions of deaths every year, and their burden is felt across every income group. Deaths from lung cancer and COPD are rising, while lower respiratory infections remain a persistent threat.
I'm pleased to share a new milestone: 3,000 citations according to the @scopus.bsky.social 👨🏻🔬✨ #biomedicalresearch #scopus
Our new paper, "Phage Therapy in People with Cystic Fibrosis: A Systematic Review," has been published (a few months ago) in the International Journal of Infectious Diseases 👨🏻🔬🦠💊 #PhageTherapy #CysticFibrosis #SystematicReview 🔗 doi.org/10.1016/j.ij...
Redirecting
doi.org
Five years later, I remain deeply grateful to Prof. Bruce Stanton for his generous praise of my sole-author paper. For those interested, the publication is available here: doi.org/10.3389/fpha... #tbt #cysticfibrosis #drugdevelopment #precisionmedicine #cftrmodulators
Even having published >70 scientific papers, I actively seek out new concepts to fuel and improve my writing skills. 📝🎖️👨🏻🔬
Our new paper has been published: "Chiral Resolution, stereochemical assignment and correction of a cystic fibrosis-causing variant of Aryl-Triazolyl-methanol derivative" in Journal of Molecular Strucuture 🧑🔬🧬💊 #medicinalchemistry #drugdevelopment #cysticfibrosis doi.org/10.1016/j.mo...
Redirecting
doi.org
🏆 I am honored to share that ScholarGPS has recognized me as a Highly Ranked Scholar (analysis of Prior Five Years) in three specialties: Precision Medicine (for the second year running), Cystic Fibrosis, and Fibrosis. 👨🏻🔬📚🧬🔬🫁 #biomedicalresearch #cysticfibrosis #precisionmedicine
Our new paper has been published: "Extracellular Vesicles: Multimodal Tools for Diagnosis, Prognosis, and Therapy in Respiratory Diseases" in Expert Review in Molecular Medicine | @universitypress.cambridge.org 🧑🔬🧬🫁 #extracellularvesicles #lungdisease #biomarker #therapydevelopment bit.ly/4nBXfCG
Extracellular Vesicles: Multimodal Tools for Diagnosis, Prognosis, and Therapy in Respiratory Diseases | Expert Reviews in Molecular Medicine | Cambridge Core
Extracellular Vesicles: Multimodal Tools for Diagnosis, Prognosis, and Therapy in Respiratory Diseases - Volume 27
cambridge.org