Precise treatment of cystic fibrosis–current treatments and perspectives for using CRISPR

TY - JOUR

T1 - Precise treatment of cystic fibrosis–current treatments and perspectives for using CRISPR

AU - Colemeadow, Josie

AU - Joyce, Holly

AU - Turcanu, Victor

PY - 2016/3/3

Y1 - 2016/3/3

N2 - Cystic fibrosis (CF) affects about 70,000 individuals worldwide, whose lives are shortened mainly due to chronic pulmonary infections resulting from impaired clearance of abnormally viscous airway mucus. The development of novel drugs targeting specific CFTR gene mutations in a precision medicine framework improved treatment, so that for patients born in 2000–2003 in the UK, the median life expectancy was estimated at around 40 years. Moreover the discovery of the CRISPR (Classes of Regularly Interspaced Palindromic Repeats) and Cas9 (Crispr-ASsociated) nuclease system opened the perspective of specifically correcting the defective CFTR gene as recently demonstrated in a model of intestinal stem cell organoids from CF patients. In the present review, we shall outline the existing state-of-art treatments and the perspectives for the precision treatment of CF opened by CRISPR.

AB - Cystic fibrosis (CF) affects about 70,000 individuals worldwide, whose lives are shortened mainly due to chronic pulmonary infections resulting from impaired clearance of abnormally viscous airway mucus. The development of novel drugs targeting specific CFTR gene mutations in a precision medicine framework improved treatment, so that for patients born in 2000–2003 in the UK, the median life expectancy was estimated at around 40 years. Moreover the discovery of the CRISPR (Classes of Regularly Interspaced Palindromic Repeats) and Cas9 (Crispr-ASsociated) nuclease system opened the perspective of specifically correcting the defective CFTR gene as recently demonstrated in a model of intestinal stem cell organoids from CF patients. In the present review, we shall outline the existing state-of-art treatments and the perspectives for the precision treatment of CF opened by CRISPR.

KW - CFTR

KW - CRISPR

KW - Cystic fibrosis

KW - F508del mutation

KW - ivacaftor

KW - lumacaftor

UR - http://www.scopus.com/inward/record.url?scp=85034710336&partnerID=8YFLogxK

U2 - 10.1080/23808993.2016.1146077

DO - 10.1080/23808993.2016.1146077

M3 - Review article

AN - SCOPUS:85034710336

SN - 2380-8993

VL - 1

SP - 169

EP - 180

JO - Expert Review of Precision Medicine and Drug Development

JF - Expert Review of Precision Medicine and Drug Development

IS - 2

ER -