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Focused ultrasound–mediated brain genome editing
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 t...
| Autores principales: | , , , , , , , , , , , , , , , , , , , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
National Academy of Sciences
2023
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10450663/ https://www.ncbi.nlm.nih.gov/pubmed/37579143 http://dx.doi.org/10.1073/pnas.2302910120 |
| _version_ | 1785095249000923136 |
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| author | Lao, Yeh-Hsing Ji, Robin Zhou, Joyce K. Snow, Kathy J. Kwon, Nancy Saville, Ethan He, Siyu Chauhan, Shradha Chi, Chun-Wei Datta, Malika S. Zhang, Hairong Quek, Chai Hoon Cai, S. Sarah Li, Mingqiang Gaitan, Yaned Bechtel, Lawrence Wu, Shih-Ying Lutz, Cathleen M. Tomer, Raju Murray, Stephen A. Chavez, Alejandro Konofagou, Elisa E. Leong, Kam W. |
| author_facet | Lao, Yeh-Hsing Ji, Robin Zhou, Joyce K. Snow, Kathy J. Kwon, Nancy Saville, Ethan He, Siyu Chauhan, Shradha Chi, Chun-Wei Datta, Malika S. Zhang, Hairong Quek, Chai Hoon Cai, S. Sarah Li, Mingqiang Gaitan, Yaned Bechtel, Lawrence Wu, Shih-Ying Lutz, Cathleen M. Tomer, Raju Murray, Stephen A. Chavez, Alejandro Konofagou, Elisa E. Leong, Kam W. |
| author_sort | Lao, Yeh-Hsing |
| collection | PubMed |
| description | 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. |
| format | Online Article Text |
| id | pubmed-10450663 |
| institution | National Center for Biotechnology Information |
| language | English |
| publishDate | 2023 |
| publisher | National Academy of Sciences |
| record_format | MEDLINE/PubMed |
| spelling | pubmed-104506632023-08-26 Focused ultrasound–mediated brain genome editing Lao, Yeh-Hsing Ji, Robin Zhou, Joyce K. Snow, Kathy J. Kwon, Nancy Saville, Ethan He, Siyu Chauhan, Shradha Chi, Chun-Wei Datta, Malika S. Zhang, Hairong Quek, Chai Hoon Cai, S. Sarah Li, Mingqiang Gaitan, Yaned Bechtel, Lawrence Wu, Shih-Ying Lutz, Cathleen M. Tomer, Raju Murray, Stephen A. Chavez, Alejandro Konofagou, Elisa E. Leong, Kam W. Proc Natl Acad Sci U S A Biological Sciences 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. National Academy of Sciences 2023-08-14 2023-08-22 /pmc/articles/PMC10450663/ /pubmed/37579143 http://dx.doi.org/10.1073/pnas.2302910120 Text en Copyright © 2023 the Author(s). Published by PNAS. https://creativecommons.org/licenses/by-nc-nd/4.0/This open access article is distributed under Creative Commons Attribution-NonCommercial-NoDerivatives License 4.0 (CC BY-NC-ND) (https://creativecommons.org/licenses/by-nc-nd/4.0/) . |
| spellingShingle | Biological Sciences Lao, Yeh-Hsing Ji, Robin Zhou, Joyce K. Snow, Kathy J. Kwon, Nancy Saville, Ethan He, Siyu Chauhan, Shradha Chi, Chun-Wei Datta, Malika S. Zhang, Hairong Quek, Chai Hoon Cai, S. Sarah Li, Mingqiang Gaitan, Yaned Bechtel, Lawrence Wu, Shih-Ying Lutz, Cathleen M. Tomer, Raju Murray, Stephen A. Chavez, Alejandro Konofagou, Elisa E. Leong, Kam W. 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 | Biological Sciences |
| url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10450663/ https://www.ncbi.nlm.nih.gov/pubmed/37579143 http://dx.doi.org/10.1073/pnas.2302910120 |
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