Beyond cystic fibrosis transmembrane conductance regulator therapy: a perspective on gene therapy and small molecule treatment for cystic fibrosis.
Journal
Gene therapy
ISSN: 1476-5462
Titre abrégé: Gene Ther
Pays: England
ID NLM: 9421525
Informations de publication
Date de publication:
09 2019
09 2019
Historique:
received:
10
09
2018
accepted:
25
06
2019
revised:
07
04
2019
pubmed:
14
7
2019
medline:
25
2
2020
entrez:
14
7
2019
Statut:
ppublish
Résumé
Cystic fibrosis (CF) is a life-limiting disease caused by defective or deficient cystic fibrosis transmembrane conductance regulator (CFTR) activity. The recent advent of the FDA-approved CFTR modulator drug ivacaftor, alone or in combination with lumacaftor or tezacaftor, has enabled treatment of the majority of patients suffering from CF. Even before the identification of the CFTR gene, gene therapy was put forward as a viable treatment option for this genetic condition. However, initial enthusiasm has been hampered as CFTR gene delivery to the lungs has proven to be more challenging than expected. This review covers the contemporary clinical and scientific knowledge base for small molecule CFTR modulator drug therapy, gene delivery vectors and CRISPR/Cas9 gene editing and highlights the prospect of these technologies for future treatment options.
Identifiants
pubmed: 31300729
doi: 10.1038/s41434-019-0092-5
pii: 10.1038/s41434-019-0092-5
doi:
Substances chimiques
Aminophenols
0
CFTR protein, human
0
Quinolones
0
Cystic Fibrosis Transmembrane Conductance Regulator
126880-72-6
ivacaftor
1Y740ILL1Z
Types de publication
Journal Article
Review
Langues
eng
Sous-ensembles de citation
IM
Pagination
354-362Commentaires et corrections
Type : CommentIn
Références
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