Focused ultrasound–mediated brain genome editing

Yeh Hsing Lao; Robin Ji; Joyce K. Zhou; Kathy J. Snow; Nancy Kwon; Ethan Saville; Siyu He; Shradha Chauhan; Chun Wei Chi; Malika S. Datta; Hairong Zhang; Chai Hoon Quek; S. Sarah Cai; Mingqiang Li; Yaned Gaitan; Lawrence Bechtel; Shih Ying Wu; Cathleen M. Lutz; Raju Tomer; Stephen A. Murray; Alejandro Chavez; Elisa E. Konofagou; Kam W. Leong

doi:10.1073/PNAS.2302910120

Focused ultrasound–mediated brain genome editing

Yeh Hsing Lao
, Robin Ji
, Joyce K. Zhou
, Kathy J. Snow
, Nancy Kwon
, Ethan Saville
, Siyu He
, Shradha Chauhan
, Chun Wei Chi
, Malika S. Datta
, Hairong Zhang
, Chai Hoon Quek
, S. Sarah Cai
, Mingqiang Li
, Yaned Gaitan
, Lawrence Bechtel
, Shih Ying Wu
, Cathleen M. Lutz
, Raju Tomer
, Stephen A. Murray

Alejandro Chavez, Elisa E. Konofagou, Kam W. Leong

Pharmaceutical Sciences

Columbia University
Jackson Laboratory
SUNY Buffalo
Sun Yat-Sen University
University of California at San Diego

Research output: Contribution to journal › Article › peer-review

29 Scopus citations

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.

Original language	English
Article number	e2302910120
Journal	Proceedings of the National Academy of Sciences of the United States of America
Volume	120
Issue number	34
DOIs	https://doi.org/10.1073/PNAS.2302910120
State	Published - 2023

Keywords

CRISPR/Cas9
blood–brain barrier
focused ultrasound
gene delivery
genome editing

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10.1073/PNAS.2302910120

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Lao, Y. H., Ji, R., Zhou, J. K., Snow, K. J., Kwon, N., Saville, E., He, S., Chauhan, S., Chi, C. W., Datta, M. S., Zhang, H., Quek, C. H., Cai, S. S., Li, M., Gaitan, Y., Bechtel, L., Wu, S. Y., Lutz, C. M., Tomer, R., ... Leong, K. W. (2023). Focused ultrasound–mediated brain genome editing. Proceedings of the National Academy of Sciences of the United States of America, 120(34), Article e2302910120. https://doi.org/10.1073/PNAS.2302910120

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title = "Focused ultrasound–mediated brain genome editing",

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.",

keywords = "CRISPR/Cas9, blood–brain barrier, focused ultrasound, gene delivery, genome editing",

author = "Lao, \{Yeh Hsing\} and Robin Ji and Zhou, \{Joyce K.\} and Snow, \{Kathy J.\} and Nancy Kwon and Ethan Saville and Siyu He and Shradha Chauhan and Chi, \{Chun Wei\} and Datta, \{Malika S.\} and Hairong Zhang and Quek, \{Chai Hoon\} and Cai, \{S. Sarah\} and Mingqiang Li and Yaned Gaitan and Lawrence Bechtel and Wu, \{Shih Ying\} and Lutz, \{Cathleen M.\} and Raju Tomer and Murray, \{Stephen A.\} and Alejandro Chavez and Konofagou, \{Elisa E.\} and Leong, \{Kam W.\}",

note = "Publisher Copyright: Copyright {\textcopyright} 2023 the Author(s). Published by PNAS.",

year = "2023",

doi = "10.1073/PNAS.2302910120",

language = "English",

volume = "120",

journal = "Proceedings of the National Academy of Sciences of the United States of America",

issn = "0027-8424",

publisher = "National Academy of Sciences",

number = "34",

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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 2023, 'Focused ultrasound–mediated brain genome editing', Proceedings of the National Academy of Sciences of the United States of America, vol. 120, no. 34, e2302910120. https://doi.org/10.1073/PNAS.2302910120

TY - JOUR

T1 - Focused ultrasound–mediated brain genome editing

AU - Lao, Yeh Hsing

AU - Ji, Robin

AU - Zhou, Joyce K.

AU - Snow, Kathy J.

AU - Kwon, Nancy

AU - Saville, Ethan

AU - He, Siyu

AU - Chauhan, Shradha

AU - Chi, Chun Wei

AU - Datta, Malika S.

AU - Zhang, Hairong

AU - Quek, Chai Hoon

AU - Cai, S. Sarah

AU - Li, Mingqiang

AU - Gaitan, Yaned

AU - Bechtel, Lawrence

AU - Wu, Shih Ying

AU - Lutz, Cathleen M.

AU - Tomer, Raju

AU - Murray, Stephen A.

AU - Chavez, Alejandro

AU - Konofagou, Elisa E.

AU - Leong, Kam W.

PY - 2023

Y1 - 2023

N2 - 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.

AB - 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.

KW - CRISPR/Cas9

KW - blood–brain barrier

KW - focused ultrasound

KW - gene delivery

KW - genome editing

UR - https://www.scopus.com/pages/publications/85168220331

U2 - 10.1073/PNAS.2302910120

DO - 10.1073/PNAS.2302910120

M3 - Article

C2 - 37579143

AN - SCOPUS:85168220331

SN - 0027-8424

VL - 120

JO - Proceedings of the National Academy of Sciences of the United States of America

JF - Proceedings of the National Academy of Sciences of the United States of America

IS - 34

M1 - e2302910120

ER -

Focused ultrasound–mediated brain genome editing

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Keywords

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