Mastodawn

Lipid #nanoparticles successfully delivered a full-length, healthy #CFTR gene into human airway cells, restoring essential biological function in a cystic #fibrosis model without the use of viral vectors.
#Medical #Nanotechnology #Nanomedicine #GeneTherapy #PulmonaryMedicine #sflorg
https://www.sflorg.com/2026/02/med02172601.html

Nanoparticle-based gene editing could expand treatment options for cystic fibrosis

Replacing the entire faulty gene rather than correcting individual mutations may allow the therapy to work across all patients with cystic fibrosis.

AP-HM - CHU de Marseille Jul 1, 2025

𝗠ieux détecter les infections pulmonaires chez les ados atteints de mucoviscidose ?
Une étude AP-HM montre l’intérêt des crachats induits (CI) dans l’analyse microbiologique.
Un travail porté par Marjory Guedes et le CRCM pédiatrique, publié dans Pediatric Pulmonology.

#Mucoviscidose #MaladiesRares #RechercheClinique #SantéRespiratoire #CFTR #Infectiologie #JeuneChercheur

Victoria Stuart 🇨🇦 🏳️‍⚧️Sep 17, 2023

Life-changing cystic fibrosis treatment wins US$3-million Breakthrough Prize
https://www.nature.com/articles/d41586-023-02890-1
Discussion: https://news.ycombinator.com/item?id=37535537

* triple-drug combination Trikafta helps 90% of people w. cystic fibrosis
* inherited disorder affecting lungs, other organs
* mutations in gene that makes the cystic fibrosis transmembrane conductance regulator protein (CFTR)
* involved in production of mucus, sweat, other fluids

#CysticFibrosis #Trikafta #disease #medicine #BreakthroughPrize #CFTR

Life-changing cystic fibrosis treatment wins US$3-million Breakthrough Prize

Trio of chemists who developed the combination drug Trikafta are among the winners of five major awards in life sciences, physics and mathematics.

Hugues Abriel Feb 28, 2023

This is the perfect day: we still need CHF 530 to reach our goal! #ScienceInAfrica #CF #CFTR
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RT @pateixidor
In our @wemakeit crowdfunding campaign we are trying to raise enough money to buy a Nanoduct® device that will improve the diagnosis of #cysticfibrosis in Africa. @AxonlabAG would you contribute in this #RareDiseaseDay2023? @SwissIonChannel @UDNIss https://wemakeit.com/projects/cystic-fibrosis-in-africa
https://twitter.com/pateixidor/status/1630494060682047488

Zystische Fibrose in Afrika – Crowdfunding bei wemakeit

Wir sind Nada und Michèle, zwei Studentinnen der Universität Bern (Prof. Abriel Labor). Wir möchten ein Testgerät zur Erkennung von Zystischer Fibrose in Afrika kaufen. Kannst du uns helfen?

Crowdfunding bei wemakeit

Hugues Abriel Dec 16, 2022

First year PhD student Nada Elmakhzen presented in Rabat her data on sequencing the complete #CFTR gene using #Nanopore technologies. Many geneticists were impressed! #AfSHG2022 #GenomicsInAfrica #ScienceInAfrica #RareDiseases
@unibern

Marco Radi Nov 21, 2022

As additional Appendix to my #introduction, I would like to #boost our recent #DrugDiscovery efforts in the field of #CysticFibrosis: we have developed #MultiTarget compounds acting at the same time as #CFTR #Correctors and #Antibacterials or #Antivirals depending on the specific functionalization of the same chemotype.

https://chemistry-europe.onlinelibrary.wiley.com/doi/10.1002/cmdc.202200277
https://pubs.acs.org/doi/10.1021/acs.jmedchem.9b01416