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Focused ultrasound-mediated brain genome editing

Gene editing in the mammalian brain has been challenging because of the restricted transport imposed by the blood-brain barrier (BBB). Current approaches rely on local injection to bypass the BBB. However, such administration is highly invasive and not amenable to treating certain delicate regions o...

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Autores principales: Leong, Kam, Lao, Yeh-Hsing, Ji, Robin, Zhou, Joyce, Snow, Kathy, Kwon, Nancy, Saville, Ethan, He, Siyu, Chauhan, Shradha, Chi, Chun-Wei, Datta, Malika, Zhang, Hairong, Quek, Chai Hoon, Cai, Sarah, Li, Mingqiang, Gaitan, Yaned, Bechtel, Lawrence, Wu, Shih-Ying, Lutz, Cathleen, Tomer, Raju, Murray, Stephen, Chavez, Alejandro, Konofagou, Elisa
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Journal Experts 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9882596/
https://www.ncbi.nlm.nih.gov/pubmed/36712096
http://dx.doi.org/10.21203/rs.3.rs-2365576/v1
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author Leong, Kam
Lao, Yeh-Hsing
Ji, Robin
Zhou, Joyce
Snow, Kathy
Kwon, Nancy
Saville, Ethan
He, Siyu
Chauhan, Shradha
Chi, Chun-Wei
Datta, Malika
Zhang, Hairong
Quek, Chai Hoon
Cai, Sarah
Li, Mingqiang
Gaitan, Yaned
Bechtel, Lawrence
Wu, Shih-Ying
Lutz, Cathleen
Tomer, Raju
Murray, Stephen
Chavez, Alejandro
Konofagou, Elisa
author_facet Leong, Kam
Lao, Yeh-Hsing
Ji, Robin
Zhou, Joyce
Snow, Kathy
Kwon, Nancy
Saville, Ethan
He, Siyu
Chauhan, Shradha
Chi, Chun-Wei
Datta, Malika
Zhang, Hairong
Quek, Chai Hoon
Cai, Sarah
Li, Mingqiang
Gaitan, Yaned
Bechtel, Lawrence
Wu, Shih-Ying
Lutz, Cathleen
Tomer, Raju
Murray, Stephen
Chavez, Alejandro
Konofagou, Elisa
author_sort Leong, Kam
collection PubMed
description Gene editing in the mammalian brain has been challenging because of the restricted transport imposed by the blood-brain barrier (BBB). Current approaches 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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spelling pubmed-98825962023-01-28 Focused ultrasound-mediated brain genome editing Leong, Kam Lao, Yeh-Hsing Ji, Robin Zhou, Joyce Snow, Kathy Kwon, Nancy Saville, Ethan He, Siyu Chauhan, Shradha Chi, Chun-Wei Datta, Malika Zhang, Hairong Quek, Chai Hoon Cai, Sarah Li, Mingqiang Gaitan, Yaned Bechtel, Lawrence Wu, Shih-Ying Lutz, Cathleen Tomer, Raju Murray, Stephen Chavez, Alejandro Konofagou, Elisa Res Sq Article Gene editing in the mammalian brain has been challenging because of the restricted transport imposed by the blood-brain barrier (BBB). Current approaches 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. American Journal Experts 2023-01-20 /pmc/articles/PMC9882596/ /pubmed/36712096 http://dx.doi.org/10.21203/rs.3.rs-2365576/v1 Text en https://creativecommons.org/licenses/by/4.0/This work is licensed under a Creative Commons Attribution 4.0 International License (https://creativecommons.org/licenses/by/4.0/) , which allows reusers to distribute, remix, adapt, and build upon the material in any medium or format, so long as attribution is given to the creator. The license allows for commercial use.
spellingShingle Article
Leong, Kam
Lao, Yeh-Hsing
Ji, Robin
Zhou, Joyce
Snow, Kathy
Kwon, Nancy
Saville, Ethan
He, Siyu
Chauhan, Shradha
Chi, Chun-Wei
Datta, Malika
Zhang, Hairong
Quek, Chai Hoon
Cai, Sarah
Li, Mingqiang
Gaitan, Yaned
Bechtel, Lawrence
Wu, Shih-Ying
Lutz, Cathleen
Tomer, Raju
Murray, Stephen
Chavez, Alejandro
Konofagou, Elisa
Focused ultrasound-mediated brain genome editing
title Focused ultrasound-mediated brain genome editing
title_full Focused ultrasound-mediated brain genome editing
title_fullStr Focused ultrasound-mediated brain genome editing
title_full_unstemmed Focused ultrasound-mediated brain genome editing
title_short Focused ultrasound-mediated brain genome editing
title_sort focused ultrasound-mediated brain genome editing
topic Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9882596/
https://www.ncbi.nlm.nih.gov/pubmed/36712096
http://dx.doi.org/10.21203/rs.3.rs-2365576/v1
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