Gene editing as a promising approach for respiratory diseases

© Article author(s) (or their employer(s) unless otherwise stated in the text of the article) 2018. All rights reserved. No commercial use is permitted unless otherwise expressly granted..

Respiratory diseases, which are leading causes of mortality and morbidity in the world, are dysfunctions of the nasopharynx, the trachea, the bronchus, the lung and the pleural cavity. Symptoms of chronic respiratory diseases, such as cough, sneezing and difficulty breathing, may seriously affect the productivity, sleep quality and physical and mental well-being of patients, and patients with acute respiratory diseases may have difficulty breathing, anoxia and even life-threatening respiratory failure. Respiratory diseases are generally heterogeneous, with multifaceted causes including smoking, ageing, air pollution, infection and gene mutations. Clinically, a single pulmonary disease can exhibit more than one phenotype or coexist with multiple organ disorders. To correct abnormal function or repair injured respiratory tissues, one of the most promising techniques is to correct mutated genes by gene editing, as some gene mutations have been clearly demonstrated to be associated with genetic or heterogeneous respiratory diseases. Zinc finger nucleases (ZFN), transcription activator-like effector nucleases (TALEN) and clustered regulatory interspaced short palindromic repeats/CRISPR-associated protein 9 (CRISPR/Cas9) systems are three innovative gene editing technologies developed recently. In this short review, we have summarised the structure and operating principles of the ZFNs, TALENs and CRISPR/Cas9 systems and their preclinical and clinical applications in respiratory diseases.

Medienart:

E-Artikel

Erscheinungsjahr:

2018

Erschienen:

2018

Enthalten in:

Zur Gesamtaufnahme - volume:55

Enthalten in:

Journal of medical genetics - 55(2018), 3 vom: 16. März, Seite 143-149

Sprache:

Englisch

Beteiligte Personen:

Bai, Yichun [VerfasserIn]
Liu, Yang [VerfasserIn]
Su, Zhenlei [VerfasserIn]
Ma, Yana [VerfasserIn]
Ren, Chonghua [VerfasserIn]
Zhao, Runzhen [VerfasserIn]
Ji, Hong-Long [VerfasserIn]

Links:

Volltext

Themen:

CRISPR/Cas9
EC 3.1.-
Gene editing
Journal Article
Respiratory diseases
Review
TALEN
Transcription Activator-Like Effector Nucleases
ZFN
Zinc Finger Nucleases

Anmerkungen:

Date Completed 20.09.2019

Date Revised 20.09.2019

published: Print-Electronic

Citation Status MEDLINE

doi:

10.1136/jmedgenet-2017-104960

funding:

Förderinstitution / Projekttitel:

PPN (Katalog-ID):

NLM27964938X