Understand CFTR mutations and their role in cystic fibrosis: https://bit.ly/41PUwO5
#CysticFibrosis #CFTR #CysticFibrosisNewsToday #Bionews
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This study by Yu-Chen Chang, Zhiwei Ma, Shi-Ting He, Yoshiro Sohma & Tzyh-Chang Hwang examines two distinct #mechanisms for GlyH-101 block of the #CFTR chloride channel 🔬 ⚡️
📜 Read the #Research: physoc.onlinelibrary.wiley.com/doi/10.1113/...
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Understand class 1 mutations: https://bit.ly/3LoYZSn
Protein production, or class 1, mutations stop the CFTR protein from being made. These include nonsense and splicing mutations, found in about 10% of people with cystic fibrosis.
#CysticFibrosis #CFTR #CysticFibrosisNewsToday #Bionews
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Translational readthrough-inducing drugs (TRIDs), highlighting NV848, shows promise in restoring #CFTR function in #cysticfibrosis patients with #nonsensevariants, offering hope for personalized therapy where current treatments fall short. bit.ly/48Ilco0 #GIMO
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Xiaolong Gao offers this #Perspective on the work of Závoti & Csanády (2025) into #molecular and pharmacological evaluation of rare, cystic fibrosis-causing missense mutations of the #CFTR channel 💭
🔗 Access it here: physoc.onlinelibrary.wiley.com/doi/10.1113/...
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In this study, Olivér Závoti & László Csanády performed #molecular and pharmacological evaluation of rare, cystic fibrosis-causing missense mutations of the #CFTR channel 🔬🧬
📜 Read the #Research here: physoc.onlinelibrary.wiley.com/doi/10.1113/...
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Exciting new study by the @trentini-lab.bsky.social #ProteinHomeostasis #RQC #CFTR
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Our new paper has been published: "Advancing cystic fibrosis treatment:
investigational agents on the horizon"
in Expert Opinion in Pharmacotherapy 👨🔬🧬🔬🧫 💊
#cysticfibrosis #CFTR #CFTRmodulator #noveltherapeutics #precisionmedicine #pharmacology #genetherapy
www.tandfonline.com/doi/full/10....
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#Moderna never-ending negative news... partner #Vertex pauses inhaled #cftr mRNA study for tolerability.
Ego too big to buy back stock on Covid windfall, instead poured tens of billions on tech that wasnt sufficiently mature.
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Novel Co-Occurrence of #Trisomy21 and Heterozygous #CFTR Mutation
onlinelibrary.wiley.com/doi/10.1002/...
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Our new paper has been published: "Recent developments in cystic fibrosis drug discovery: where are we today?"
in Expert Opinion in Drug Discovery Taylor & Francis 👨🔬🧬🔬🧫 💊
#cysticfibrosis #CFTR #CFTRmodulator #noveltherapeutics #precisionmedicine
#multiomics
www.tandfonline.com/eprint/RNWZS...
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Personalised #CFTR Modulator Treatment Initiation and Monitoring in CF-Related Liver Disease: When Less Is More
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Karen Raraigh is a #CFTR superstar and our latest highlight in the #Unique role series. Learn about her career, which she has largely dedicated to #cysticfibrosis research and education. #genechat #precisionmedicine
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Our new paper has been published: "Cystic fibrosis: new challenges and perspectives beyond elexacaftor/tezacaftor/ivacaftor" in Therapeutic Advances in Respiratory Disease 👨🔬🧬🔬🧫 💊
#cysticfibrosis #CFTR #CFTRmodulator #noveltherapeutics #precisionmedicine
journals.sagepub.com/doi/10.1177/...
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Screenshot of Table 1: Quality control metrics of long-read (PacBio Revio) and short-read (Illumina Novaseq) genome sequencing data of cystic fibrosis patient samples Variant counts are based on regions defined by the genome-in-a-bottle consortium. bp, base pairs; CF1, whole-blood sample; CF2, newborn dried blood spot sample; DBS, dried blood spot; Gb, gigabases; het/nonref-hom, heterozygosity to nonreference homozygosity ratio; indels, insertions and deletions; KB, kilobases; M, million; SNPs, single-nucleotide polymorphisms; Ti/Tv, transition/transversion ratio.
Long-read sequencing phases rare pathogenic variants from minimal DNA, enabling faster newborn disease screening without parental testing bit.ly/3D4IkA3 #GIMO #CFTR #LongReadSequencing
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Francis Collins, whose lab discovered the #CFTr gene leading to transformative therapies in #cysticfibrosis
Sad day for science 😔
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"Some scientists search for a solution to a problem and then move on, other will just keep digging". meeting organizer Gergely Szakacs about our evening plenary speaker, who will talk about a channel at a transporter meeting 😉
#CFTR #ABC2025
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Our latest case study highlights how Frédéric Becq from the University of Poitiers uses automated patch-clamp technology to deepen our understanding of CFTR function and its interactions with drugs.
Read more here: www.nanion.de/wp-content/u...
#ionchannels #CFTR
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Our new paper has been published: "Global Functional Genomics Reveals GRK5 as a Cystic Fibrosis Therapeutic Target Synergistic with Current Modulators" in @cp-iscience.bsky.social 👨🔬🧬🔬🧫 💊
#cysticfibrosis #CFTR #FunctionalGenomics #noveltherapeutics #precisionmedicine www.cell.com/iscience/ful...
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How long till the first wave of diabetic nephropathy starts hitting the Adult #CysticFibrosis clinics? The new era of highly effective #cftr modulators bring a bundle of ‘regular’ maladies to CFers #ElexacaftorTezacaftorIvacaftor #alyftrek #deutivacaftor #lumacaftor #ivacaftor
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#tbt Imagine you are in the audience to watch the plenary session at NACFC and a full figure of your sole author article is used by the speaker to talk about CFTR biology and modulation... what a memory! ❣️👨🏻🔬🧬
#nacfc2022 #cysticfibrosis #cftr #personalizedmedicine #timehop
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