Cystic fibrosis precision therapeutics: emerging considerations

D Joshi, A Ehrhardt, JS Hong… - Pediatric …, 2019 - Wiley Online Library
… ” interventions for specific abnormalities of the cystic fibrosis … with CF worldwide—barriers
to precision therapy remain. These … has occurred regarding CF precision medicine, this review …

Cystic fibrosis–an example of personalized and precision medicine

M Skov, CR Hansen, T Pressler - Apmis, 2019 - Wiley Online Library
Cystic fibrosis (CF) is a severe, monogenic, autosomal recessive disease caused by
mutations in the CFTR (cystic fibrosis transmembrane regulator) gene, where disturbed chloride …

[HTML][HTML] Recent progress in translational cystic fibrosis research using precision medicine strategies

DM Cholon, M Gentzsch - Journal of Cystic Fibrosis, 2018 - Elsevier
… in developing precision therapies for cystic fibrosis; however, highly effective treatments that
target … drug efficacy to support individualized treatment plans and translational research are …

Making precision medicine personal for cystic fibrosis

C Manfredi, JM Tindall, JS Hong, EJ Sorscher - Science, 2019 - science.org
… It was originally anticipated that well-established disease subclasses would serve as an
organizing principle for precision CF treatments and help identify specific compounds for …

[HTML][HTML] CFTR modulators: the changing face of cystic fibrosis in the era of precision medicine

M Lopes-Pacheco - Frontiers in pharmacology, 2020 - frontiersin.org
Cystic fibrosis (CF) is a lethal inherited disease caused by mutations in the CF … Mutations
in the cystic fibrosis transmembrane conductance regulator (CFTR) gene cause cystic fibrosis (…

Cystic fibrosis in the era of precision medicine

SM Paranjape, PJ Mogayzel Jr - Paediatric respiratory reviews, 2018 - Elsevier
… The treatment of people with cystic fibrosis (CF) has been … embodies the goals of precision
medicine, which incorporate … individualized ushering in a new era of precision medicine. …

Progress in precision medicine in cystic fibrosis: a focus on CFTR modulator therapy

DH Tewkesbury, RC Robey, PJ Barry - Breathe, 2021 - Eur Respiratory Soc
… The genetic multisystem condition cystic fibrosis (CF) has seen a paradigm shift in … , treatment
advances had focused on the downstream consequences of a dysfunctional cystic fibrosis

Correlating cystic fibrosis transmembrane conductance regulator function with clinical features to inform precision treatment of cystic fibrosis

AF McCague, KS Raraigh, MJ Pellicore… - American journal of …, 2019 - atsjournals.org
… of precision treatment for cystic fibrosis using small-molecule therapeutics has created a
need to estimate potential clinical improvements attributable to increases in cystic fibrosis

Precision medicine based on CFTR genotype for people with cystic fibrosis

I Haq, M Almulhem, S Soars, D Poulton… - Pharmacogenomics …, 2022 - Taylor & Francis
cystic fibrosis transmembrane conductance regulator gene. This causes multisystem disease
due to dysfunction of the cystic fibrosis … Until recently, treatment was directed at managing …

[HTML][HTML] CFTR modulators: shedding light on precision medicine for cystic fibrosis

M Lopes-Pacheco - Frontiers in pharmacology, 2016 - frontiersin.org
… Pathophysiological cascade of respiratory disorder in cystic fibrosis – Cellular mechanism
of cystic fibrosis begins with the defective CFTR (cystic fibrosis transmembrane conductance …