Progress and Perspective of CRISPR-Cas9 Technology in Translational Medicine.
clustered regularly interspaced short palindromic repeats-associated protein 9 (CRISPR-Cas9)
gene regulation
gene therapy
translational medicine
Journal
Advanced science (Weinheim, Baden-Wurttemberg, Germany)
ISSN: 2198-3844
Titre abrégé: Adv Sci (Weinh)
Pays: Germany
ID NLM: 101664569
Informations de publication
Date de publication:
09 2023
09 2023
Historique:
revised:
29
03
2023
received:
09
01
2023
medline:
6
9
2023
pubmed:
25
6
2023
entrez:
25
6
2023
Statut:
ppublish
Résumé
Translational medicine aims to improve human health by exploring potential treatment methods developed during basic scientific research and applying them to the treatment of patients in clinical settings. The advanced perceptions of gene functions have remarkably revolutionized clinical treatment strategies for target agents. However, the progress in gene editing therapy has been hindered due to the severe off-target effects and limited editing sites. Fortunately, the development in the clustered regularly interspaced short palindromic repeats associated protein 9 (CRISPR-Cas9) system has renewed hope for gene therapy field. The CRISPR-Cas9 system can fulfill various simple or complex purposes, including gene knockout, knock-in, activation, interference, base editing, and sequence detection. Accordingly, the CRISPR-Cas9 system is adaptable to translational medicine, which calls for the alteration of genomic sequences. This review aims to present the latest CRISPR-Cas9 technology achievements and prospect to translational medicine advances. The principle and characterization of the CRISPR-Cas9 system are firstly introduced. The authors then focus on recent pre-clinical and clinical research directions, including the construction of disease models, disease-related gene screening and regulation, and disease treatment and diagnosis for multiple refractory diseases. Finally, some clinical challenges including off-target effects, in vivo vectors, and ethical problems, and future perspective are also discussed.
Identifiants
pubmed: 37356052
doi: 10.1002/advs.202300195
pmc: PMC10477906
doi:
Types de publication
Journal Article
Review
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
e2300195Subventions
Organisme : Key Research and Development Plan of Zhejiang Province "Spearhead"
ID : 2022C03007
Organisme : Wenzhou Major Science and Technology Innovation Tackling Project
ID : 2020ZY0036
Organisme : Zhejiang Provincial Natural Science Foundation of China
ID : LGF22C100003
Organisme : Wenzhou high-level innovation team : Development and application team of functional liver cancer-on-a-chip program of Wenzhou Institute, University of Chinese Academy of Sciences
ID : WIUCASQD2021012
Informations de copyright
© 2023 The Authors. Advanced Science published by Wiley-VCH GmbH.
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