CFTR: cystic fibrosis and beyond
MA Mall, D Hartl - 2014 - Eur Respiratory Soc
Cystic fibrosis (CF) remains the most common fatal hereditary lung disease. The discovery of
the cystic fibrosis transmembrane conductance regulator (CFTR) gene 25 years ago set the …
the cystic fibrosis transmembrane conductance regulator (CFTR) gene 25 years ago set the …
Cystic fibrosis: emergence of highly effective targeted therapeutics and potential clinical implications
MA Mall, N Mayer-Hamblett… - American journal of …, 2020 - atsjournals.org
Cystic fibrosis (CF) remains the most common life-shortening hereditary disease in white
populations, with high morbidity and mortality related to chronic airway mucus obstruction …
populations, with high morbidity and mortality related to chronic airway mucus obstruction …
Cystic fibrosis: exploiting its genetic basis in the hunt for new therapies
JL Kreindler - Pharmacology & therapeutics, 2010 - Elsevier
Cystic fibrosis (CF) is caused by mutations in the gene encoding the cystic fibrosis
transmembrane conductance regulator (CFTR), an anion channel expressed in epithelial …
transmembrane conductance regulator (CFTR), an anion channel expressed in epithelial …
Current and future treatment options for cystic fibrosis lung disease: latest evidence and clinical implications
C Edmondson, JC Davies - Therapeutic advances in chronic …, 2016 - journals.sagepub.com
Treatment for cystic fibrosis (CF) has conventionally targeted downstream consequences of
the defect such as mucus plugging and infection. More recently, significant advances have …
the defect such as mucus plugging and infection. More recently, significant advances have …
[HTML][HTML] Challenges facing airway epithelial cell-based therapy for cystic fibrosis
A Berical, RE Lee, SH Randell… - Frontiers in pharmacology, 2019 - frontiersin.org
Mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene cause
the life-limiting hereditary disease, cystic fibrosis (CF). Decreased or absent functional CFTR …
the life-limiting hereditary disease, cystic fibrosis (CF). Decreased or absent functional CFTR …
The future of cystic fibrosis treatment: from disease mechanisms to novel therapeutic approaches
SY Graeber, MA Mall - The Lancet, 2023 - thelancet.com
With the 2019 breakthrough in the development of highly effective modulator therapy
providing unprecedented clinical benefits for over 90% of patients with cystic fibrosis who …
providing unprecedented clinical benefits for over 90% of patients with cystic fibrosis who …
Future directions in early cystic fibrosis lung disease research: an NHLBI workshop report
BW Ramsey, S Banks-Schlegel, FJ Accurso… - American journal of …, 2012 - atsjournals.org
Since the 1989 discovery that mutations in the cystic fibrosis transmembrane conductance
regulator (CFTR) gene cause cystic fibrosis (CF), there has been substantial progress …
regulator (CFTR) gene cause cystic fibrosis (CF), there has been substantial progress …
[HTML][HTML] Emerging therapeutic approaches for cystic fibrosis. From gene editing to personalized medicine
I Pranke, A Golec, A Hinzpeter, A Edelman… - Frontiers in …, 2019 - frontiersin.org
An improved understanding of the cystic fibrosis (CF) transmembrane conductance regulator
(CFTR) protein structure and the consequences of CFTR gene mutations have allowed the …
(CFTR) protein structure and the consequences of CFTR gene mutations have allowed the …
Novel personalized therapies for cystic fibrosis: treating the basic defect in all patients
MD Amaral - Journal of internal medicine, 2015 - Wiley Online Library
Cystic fibrosis (CF) is the most common genetic life‐shortening condition in Caucasians.
Despite being a multi‐organ disease, CF is classically diagnosed by symptoms of …
Despite being a multi‐organ disease, CF is classically diagnosed by symptoms of …
A new era in the treatment of cystic fibrosis: correction of the underlying CFTR defect
MP Boyle, K De Boeck - The Lancet Respiratory Medicine, 2013 - thelancet.com
Cystic fibrosis is caused by dysfunction or deficiency of the cystic fibrosis transmembrane
conductance regulator (CFTR) protein, an epithelial chloride channel that has a key role in …
conductance regulator (CFTR) protein, an epithelial chloride channel that has a key role in …