Gene editing therapeutics based on mRNA delivery.
Base editing
CRISPR
Delivery
Gene editing
Gene therapy
Nanoparticle
mRNA
Journal
Advanced drug delivery reviews
ISSN: 1872-8294
Titre abrégé: Adv Drug Deliv Rev
Pays: Netherlands
ID NLM: 8710523
Informations de publication
Date de publication:
09 2023
09 2023
Historique:
received:
30
04
2023
revised:
20
07
2023
accepted:
26
07
2023
medline:
28
8
2023
pubmed:
30
7
2023
entrez:
29
7
2023
Statut:
ppublish
Résumé
The field of gene editing has received much attention in recent years due to its immense therapeutic potential. In particular, gene editing therapeutics, such as the CRISPR-Cas systems, base editors, and other emerging gene editors, offer the opportunity to address previously untreatable disorders. This review aims to summarize the therapeutic applications of gene editing based on mRNA delivery. We introduce gene editing therapeutics using mRNA and focus on engineering and improvement of gene editing technology. We subsequently examine ex vivo and in vivo gene editing techniques and conclude with an exploration of the next generation of CRISPR and base editing systems.
Identifiants
pubmed: 37516409
pii: S0169-409X(23)00341-1
doi: 10.1016/j.addr.2023.115026
pii:
doi:
Substances chimiques
RNA, Messenger
0
Types de publication
Journal Article
Review
Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
115026Subventions
Organisme : NIMH NIH HHS
ID : R01 MH125979
Pays : United States
Organisme : NIAID NIH HHS
ID : UM1 AI164559
Pays : United States
Organisme : NIBIB NIH HHS
ID : R01 EB029320
Pays : United States
Organisme : NIDA NIH HHS
ID : R61 DA048444
Pays : United States
Organisme : NINDS NIH HHS
ID : UG3 NS115599
Pays : United States
Informations de copyright
Copyright © 2023. Published by Elsevier B.V.
Déclaration de conflit d'intérêts
Declaration of Competing Interest The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper.