[HTML][HTML] 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 …

Disease modifying genes in cystic fibrosis: therapeutic option or one-way road?

R Büscher, H Grasemann - Naunyn-Schmiedeberg's archives of …, 2006 - Springer
Cystic fibrosis (CF) is the most common genetic disease among Caucasians and is caused
by mutations in the CF transmembrane conductance regulator (CFTR) gene. CF affects …

[HTML][HTML] Genetic influences on cystic fibrosis lung disease severity

CA Weiler, ML Drumm - Frontiers in pharmacology, 2013 - frontiersin.org
Understanding the causes of variation in clinical manifestations of disease should allow for
design of new or improved therapeutic strategies to treat the disease. If variation is caused …

Update on gene modifiers in cystic fibrosis

JM Collaco, GR Cutting - Current opinion in pulmonary medicine, 2008 - journals.lww.com
Several genes have been demonstrated to affect disease severity in CF. Furthermore, it is
likely that gene–gene and gene–environment interactions can explain a substantial portion …

Modifier gene studies to identify new therapeutic targets in cystic fibrosis

R Dorfman - Current pharmaceutical design, 2012 - ingentaconnect.com
Since the discovery of the CFTR gene mutations which cause cystic fibrosis (CF) in 1989 the
average life expectancy of CF patients has almost doubled and now exceeds 37 years. The …

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
therapeutic approaches within the past decade. Since the first clinical descriptions in the …

Gene modifiers of cystic fibrosis lung disease: A systematic review

S Shanthikumar, MN Neeland, R Saffery… - Pediatric …, 2019 - Wiley Online Library
Background Lung disease is the major source of morbidity and mortality in cystic fibrosis
(CF), with large variability in severity between patients. Although accurate prediction of lung …

Personalized medicine in CF: from modulator development to therapy for cystic fibrosis patients with rare CFTR mutations

M Harutyunyan, Y Huang, KS Mun… - … of Physiology-Lung …, 2018 - journals.physiology.org
Cystic fibrosis (CF) is the most common life-shortening genetic disease affecting~ 1 in 3,500
of the Caucasian population. CF is caused by mutations in the CF transmembrane …

CFTR modulator therapy: transforming the landscape of clinical care in cystic fibrosis

JL Taylor-Cousar, PD Robinson, M Shteinberg… - The Lancet, 2023 - thelancet.com
Following discovery of the cystic fibrosis transmembrane conductance regulator (CFTR)
gene in 1989 and subsequent elucidation of the varied CFTR protein abnormalities that …

Lung disease modifier genes in cystic fibrosis

L Guillot, J Beucher, O Tabary, P Le Rouzic… - The international journal …, 2014 - Elsevier
Cystic fibrosis (CF) is recognized as a single gene disorder. However, a considerable
diversity in its clinical phenotype has been documented since the description of the disease …