In this section
@inbook{Davies:2019:10.1016/B978-0-323-44887-1.00053-5,
author = {Davies, G and Griesenbach, U and Alton, E and Davies, JC},
booktitle = {Kendig's Disorders of the Respiratory Tract in Children},
doi = {10.1016/B978-0-323-44887-1.00053-5},
pages = {800--811.e3},
title = {53 - Molecular Therapies for Cystic Fibrosis},
url = {http://dx.doi.org/10.1016/B978-0-323-44887-1.00053-5},
year = {2019}
}
TY - CHAP
AB - This chapter describes the therapeutic strategies for cystic fibrosis which are based on targeting cystic fibrosis transmembrane conductance regulator (CFTR), either at the gene or protein level. We provide updates on small molecule CFTR modulators and gene therapy, focusing on clinical development and evaluation. The field has seen significant progress over recent years, particularly with the CFTR potentiator, ivacaftor, in patients with class III mutations. Increased understanding of the abnormalities in the structure and function of CFTR protein will help optimize the approaches required for normalizing function and, in doing so, aid the rational design of clinical trials-both in terms of the development of more efficacious drugs and the selection of appropriate patient populations. While progress with gene therapy remains some way behind, potential benefits (including being mutation agnostic and a nonsystemic route of delivery) remain significant. It may be that future optimal approaches will harness the benefits of more than one of these approaches and lead to considerable synergy. The ultimate goal for molecular and advanced therapies in cystic fibrosis is to find drugs or combinations of drugs capable of restoring CFTR function, applicable to patients with any genetic mutation.
AU - Davies,G
AU - Griesenbach,U
AU - Alton,E
AU - Davies,JC
DO - 10.1016/B978-0-323-44887-1.00053-5
EP - 811
PY - 2019///
SP - 800
TI - 53 - Molecular Therapies for Cystic Fibrosis
T1 - Kendig's Disorders of the Respiratory Tract in Children
UR - http://dx.doi.org/10.1016/B978-0-323-44887-1.00053-5
ER -
