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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.
Afiliação
  • Lao YH; Department of Biomedical Engineering, Columbia University, New York, NY 10027.
  • Ji R; Department of Pharmaceutical Sciences, University at Buffalo, The State University of New York, Buffalo, NY 14214.
  • Zhou JK; Department of Biomedical Engineering, Columbia University, New York, NY 10027.
  • Snow KJ; Department of Biomedical Engineering, Columbia University, New York, NY 10027.
  • Kwon N; The Jackson Laboratory, Bar Harbor, ME 04609.
  • Saville E; Department of Biomedical Engineering, Columbia University, New York, NY 10027.
  • He S; The Jackson Laboratory, Bar Harbor, ME 04609.
  • Chauhan S; Department of Biomedical Engineering, Columbia University, New York, NY 10027.
  • Chi CW; Department of Biological Sciences, Columbia University, New York, NY 10027.
  • Datta MS; Department of Pharmaceutical Sciences, University at Buffalo, The State University of New York, Buffalo, NY 14214.
  • Zhang H; Department of Biological Sciences, Columbia University, New York, NY 10027.
  • Quek CH; Department of Biomedical Engineering, Columbia University, New York, NY 10027.
  • Cai SS; Department of Biomedical Engineering, Columbia University, New York, NY 10027.
  • Li M; Department of Biomedical Engineering, Columbia University, New York, NY 10027.
  • Gaitan Y; Department of Biomedical Engineering, Columbia University, New York, NY 10027.
  • Bechtel L; The Third Affiliated Hospital, Sun Yat-sen University, Guangzhou 510630, China.
  • Wu SY; The Jackson Laboratory, Bar Harbor, ME 04609.
  • Lutz CM; The Jackson Laboratory, Bar Harbor, ME 04609.
  • Tomer R; Department of Biomedical Engineering, Columbia University, New York, NY 10027.
  • Murray SA; The Jackson Laboratory, Bar Harbor, ME 04609.
  • Chavez A; Department of Biological Sciences, Columbia University, New York, NY 10027.
  • Konofagou EE; The Jackson Laboratory, Bar Harbor, ME 04609.
  • Leong KW; Department of Pediatrics, University of California San Diego, La Jolla, CA 92037.
Proc Natl Acad Sci U S A ; 120(34): e2302910120, 2023 08 22.
Article em En | MEDLINE | ID: mdl-37579143
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.
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Encéfalo / Edição de Genes Idioma: En Revista: Proc Natl Acad Sci U S A Ano de publicação: 2023 Tipo de documento: Article

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Encéfalo / Edição de Genes Idioma: En Revista: Proc Natl Acad Sci U S A Ano de publicação: 2023 Tipo de documento: Article