Focused ultrasound-mediated brain genome editing.
Proc Natl Acad Sci U S A
; 120(34): e2302910120, 2023 08 22.
Article
em En
| MEDLINE
| ID: mdl-37579143
ABSTRACT
Gene editing in the brain has been challenging because of the restricted transport imposed by the blood-brain barrier (BBB). Current approaches mainly rely on local injection to bypass the BBB. However, such administration is highly invasive and not amenable to treating certain delicate regions of the brain. We demonstrate a safe and effective gene editing technique by using focused ultrasound (FUS) to transiently open the BBB for the transport of intravenously delivered CRISPR/Cas9 machinery to the brain.
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Base de dados:
MEDLINE
Assunto principal:
Encéfalo
/
Edição de Genes
Idioma:
En
Ano de publicação:
2023
Tipo de documento:
Article