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Updated: Jul 19, 2025
Focused Ultrasound Induced Blood-Brain Barrier Opening for Targeting Brain Structures and Evaluating Chemogenetic Neuromodulation
Published on: December 22, 2020
Focused ultrasound-mediated brain genome editing
Yeh-Hsing Lao1,2, Robin Ji1, Joyce K Zhou1
1Department of Biomedical Engineering, Columbia University, New York, NY 10027.
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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