Focused ultrasound gene delivery for the treatment of neurological disorders.
blood–brain barrier
focused ultrasound
gene therapy
neurological disorders
Journal
Trends in molecular medicine
ISSN: 1471-499X
Titre abrégé: Trends Mol Med
Pays: England
ID NLM: 100966035
Informations de publication
Date de publication:
Mar 2024
Mar 2024
Historique:
received:
27
10
2023
revised:
11
12
2023
accepted:
15
12
2023
medline:
18
3
2024
pubmed:
13
1
2024
entrez:
12
1
2024
Statut:
ppublish
Résumé
The transformative potential of gene therapy has been demonstrated in humans. However, there is an unmet need for non-invasive targeted gene delivery and regulation in the treatment of brain disorders. Transcranial focused ultrasound (FUS) has gained tremendous momentum to address these challenges. FUS non-invasively modulates brain cells and their environment, and is a powerful tool to facilitate gene delivery across the blood-brain barrier (BBB) with millimeter precision and promptly regulate transgene expression. This review highlights technical aspects of FUS-mediated gene therapies for the central nervous system (CNS) and lessons learned from discoveries in other organs. Understanding the possibilities and remaining obstacles of FUS-mediated gene therapy will be necessary to harness remarkable technologies and create life-changing treatments for neurological disorders.
Identifiants
pubmed: 38216449
pii: S1471-4914(23)00285-X
doi: 10.1016/j.molmed.2023.12.006
pii:
doi:
Types de publication
Journal Article
Review
Langues
eng
Sous-ensembles de citation
IM
Pagination
263-277Informations de copyright
Copyright © 2023 Elsevier Ltd. All rights reserved.
Déclaration de conflit d'intérêts
Declaration of interests The authors declare no competing interests.