학술논문
Focused ultrasound-mediated brain genome editing.
Document Type
Article
Author
Yeh-Hsing Lao; Robin Ji; Zhou, Joyce K.; Snow, Kathy J.; Kwon, Nancy; Saville, Ethan; Siyu He; Chauhan, Shradha; Chun-Wei Chi; Datta, Malika S.; Hairong Zhang; Chai Hoon Quek; Cai, S. Sarah; Mingqiang Li; Gaitan, Yaned; Bechtel, Lawrence; Shih-Ying Wu; Lutz, Cathleen M.; Tomer, Raju; Murray, Stephen A.
Source
Subject
*GENOME editing
*BLOOD-brain barrier
*CRISPRS
*
*
Language
ISSN
0027-8424
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. [ABSTRACT FROM AUTHOR]