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 …

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 (…

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 …

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 …

Cystic fibrosis lung disease modifiers and their relevance in the new era of precision medicine

A Sepahzad, DJ Morris-Rosendahl, JC Davies - Genes, 2021 - mdpi.com
… Our understanding of cystic fibrosis (CF) has grown exponentially since the discovery of
the cystic fibrosis transmembrane conductance regulator (CFTR) gene in 1989. With evolving …

The future of cystic fibrosis treatment: from disease mechanisms to novel therapeutic approaches

SY Graeber, MA Mall - The Lancet, 2023 - thelancet.com
… with cystic fibrosis who are genetically eligible for treatment, … the pathogenesis of cystic
fibrosis, working together with a … of the lungs of people with cystic fibrosis. We review progress in (…

The influence of CFTR complex alleles on precision therapy of cystic fibrosis

B Chevalier, A Hinzpeter - Journal of Cystic Fibrosis, 2020 - Elsevier
… propose adequate treatments. Conversely, cis-variants can affect treatment response when
… We wish to thank the French cystic fibrosis association Vaincre la mucoviscidose for funding …