Focused ultrasound-mediated brain genome editing.
basic_science · Level V
Where this comes from
- Record sourced from PubMed, PMID 37579143.
- Also identified by DOI 10.1073/pnas.2302910120 and PMC identifier 10450663.
- Licence recorded as CC BY-NC-ND.
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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.
Medical subject headings
- Gene Editing
- Brain