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 …

CFTR targeted therapies: recent advances in cystic fibrosis and possibilities in other diseases of the airways

SD Patel, TR Bono, SM Rowe… - European Respiratory …, 2020 - Eur Respiratory Soc
Cystic fibrosis transmembrane conductance regulator (CFTR) is an ion transporter that
regulates mucus hydration, viscosity and acidity of the airway epithelial surface. Genetic …

Progress in therapies for cystic fibrosis

K De Boeck, MD Amaral - The Lancet Respiratory Medicine, 2016 - thelancet.com
Standard follow-up and symptomatic treatment have allowed most patients with cystic
fibrosis to live to young adulthood. However, many patients still die prematurely from …

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 …

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 …

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 …

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 …

The effect of CFTR modulators on airway infection in cystic fibrosis

C Harvey, S Weldon, S Elborn, DG Downey… - International Journal of …, 2022 - mdpi.com
The advent of Cystic fibrosis transmembrane receptor (CFTR) modulators in 2012 was a
critical event in the history of cystic fibrosis (CF) treatment. Unlike traditional therapies that …

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 …

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 …