Publication: Focused ultrasound-mediated brain genome editing.
Focused ultrasound-mediated brain genome editing.
Lao YH , Ji R , Zhou JK , Snow KJ , Kwon N , Saville E , He S , Chauhan S , Chi CW , Datta MS , Zhang H , Quek CH , Cai SS , Li M , Gaitan Y , Bechtel L , Wu SY , Lutz CM , Tomer R , Murray SA , Chavez A , Konofagou EE , Leong KW
PUBMED: 37579143;
PMC: PMC10450663;
DOI: 10.1073/pnas.2302910120;
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.
Mesh Terms: [Gene Editing, Brain, Blood-Brain Barrier, Biological Transport, Microbubbles]
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.
Mesh Terms: [Gene Editing, Brain, Blood-Brain Barrier, Biological Transport, Microbubbles]
Projects & Experiments
This Publication: Focused ultrasound-mediated brain genome editing. is being used ...| Project | Initiative | Contact PI | |
|---|---|---|---|
| Delivery Systems |
Kam W Leong, PhD
(null) |
|