Gene therapy for cystic fibrosis: Challenges and prospects

H Sui, X Xu, Y Su, Z Gong, M Yao, X Liu… - Frontiers in …, 2022 - frontiersin.org
Cystic fibrosis (CF) is a life-threatening autosomal-recessive disease caused by mutations in
a single gene encoding cystic fibrosis transmembrane conductance regulator (CFTR). CF …

[PDF][PDF] The impact of highly effective modulator therapy on cystic fibrosis microbiology and inflammation

LJ Caverly, SA Riquelme, KB Hisert - Clinics in chest medicine, 2022 - Elsevier
Highly-effective cystic fibrosis transmembrane conductance regulator (CFTR) modulator
therapy (HEMT) corrects the underlying molecular defect causing cystic fibrosis (CF) …

Early human fetal lung atlas reveals the temporal dynamics of epithelial cell plasticity

H Quach, S Farrell, MJM Wu, K Kanagarajah… - Nature …, 2024 - nature.com
Studying human fetal lungs can inform how developmental defects and disease states alter
the function of the lungs. Here, we sequenced> 150,000 single cells from 19 healthy human …

[HTML][HTML] Fetal drug exposure after maternally administered CFTR modulators Elexacaftor/Tezacaftor/Ivacaftor in a rat model

D Li, Y Zhu, M Donnelley, D Parsons… - Biomedicine & …, 2024 - Elsevier
Background The potential effects of the very effective cystic fibrosis triple combination drug,
Elexacaftor/Tezacaftor/Ivacaftor (ETI) in pregnancy on prenatal development of offspring …

Therapeutic potential of phytochemicals for cystic fibrosis

H Baharara, P Kesharwani, TP Johnston… - Biofactors, 2023 - Wiley Online Library
The aim of this review was to review and discuss various phytochemicals that exhibit
beneficial effects on mutated membrane channels, and hence, improve transmembrane …

Adaptive laboratory evolution of Rhodococcus rhodochrous DSM6263 for chlorophenol degradation under hypersaline condition

J Zheng, Z Zhang, J An, Y Xue, B Yu - Microbial Cell Factories, 2023 - Springer
Background Normally, a salt amount greater than 3.5%(w/v) is defined as hypersaline. Large
amounts of hypersaline wastewater containing organic pollutants need to be treated before …

Modeling lung cell development using human pluripotent stem cells

SY Ngan, H Quach, J Dierolf, O Laselva, JA Lee… - bioRxiv, 2021 - biorxiv.org
Human PSC (hPSC) differentiations can capture developmental phenotypes and processes
and are useful for studying fundamental biological mechanisms driving tissue …

End Stage Kidney Disease in Cystic Fibrosis

ML Graber, TA MacKenzie, M Stevanovic… - medRxiv, 2024 - medrxiv.org
Background Cystic fibrosis (CF) is a multisystem autosomal recessive disease caused by
mutations in the cystic fibrosis transduction regulator (CFTR). The CFTR is expressed in all …

[PDF][PDF] Investigating the Cause and Focusing on Treating Heart Failure-Related Target Organ Damage

L Vanherle - 2023 - portal.research.lu.se
Over the last decades, survival rates after myocardial infarction (MI) have improved
significantly, resulting in increasing numbers of patients that develop chronic heart failure …

The Impact of Highly Effective Modulator Therapy Microbiology on Cystic and Inflammation Fibrosis

LJ Caverly - Advances in Cystic Fibrosis, An Issue of Clinics in …, 2022 - books.google.com
Cystic fibrosis (CF) airway disease results from the complex interactions of 3 pathologic
processes:(1) primary airway epithelial cell deficiency in CF transmembrane conductance …