Focused ultrasound–mediated brain genome editing-Reference-Cited by-同舟云学术

Focused ultrasound–mediated brain genome editing

Published:2023-08-14 Issue:34 Volume:120 Page:
ISSN:0027-8424
Container-title:Proceedings of the National Academy of Sciences
language:en
Short-container-title:Proc. Natl. Acad. Sci. U.S.A.

Author:

Lao Yeh-Hsing¹²,Ji Robin¹^ORCID,Zhou Joyce K.¹,Snow Kathy J.³^ORCID,Kwon Nancy¹^ORCID,Saville Ethan³,He Siyu¹,Chauhan Shradha⁴,Chi Chun-Wei²,Datta Malika S.⁴,Zhang Hairong¹,Quek Chai Hoon¹^ORCID,Cai S. Sarah¹^ORCID,Li Mingqiang¹⁵,Gaitan Yaned³^ORCID,Bechtel Lawrence³^ORCID,Wu Shih-Ying¹,Lutz Cathleen M.³^ORCID,Tomer Raju⁴,Murray Stephen A.³^ORCID,Chavez Alejandro⁶,Konofagou Elisa E.¹⁷^ORCID,Leong Kam W.¹⁸^ORCID

Affiliation:

1. Department of Biomedical Engineering, Columbia University, New York, NY 10027

2. Department of Pharmaceutical Sciences, University at Buffalo, The State University of New York, Buffalo, NY 14214

3. The Jackson Laboratory, Bar Harbor, ME 04609

4. Department of Biological Sciences, Columbia University, New York, NY 10027

5. The Third Affiliated Hospital, Sun Yat-sen University, Guangzhou 510630, China

6. Department of Pediatrics, University of California San Diego, La Jolla, CA 92037

7. Department of Radiology, Columbia University Medical Center, New York, NY 10032

8. Department of Systems Biology, Columbia University Medical Center, New York, NY 10032

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.

Funder

HHS | NIH | National Institute of Neurological Disorders and Stroke

HHS | NIH | NIH Office of the Director

American Society of Gene & Cell Therapy

Publisher

Proceedings of the National Academy of Sciences

Subject

Multidisciplinary

Link

https://pnas.org/doi/pdf/10.1073/pnas.2302910120

Reference16 articles.

1. The next generation of CRISPR–Cas technologies and applications