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Focused ultrasound expands intra-CSF AAV delivery to deep brain regions in rats and non-human primates.

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Title: Focused ultrasound expands intra-CSF AAV delivery to deep brain regions in rats and non-human primates.
Authors: Kofoed RH; Department of Neurosurgery, Department of Clinical Medicine, Aarhus University, 8200 Aarhus N, Denmark; Center for Experimental Neuroscience - CENSE, Department of Neurosurgery, Aarhus University Hospital, 8200 Aarhus N, Denmark; Biological Sciences, Hurvitz Brain Sciences Research Program, Sunnybrook Research Institute, Toronto, ON M4N 3M5, Canada. Electronic address: rhko@clin.au.dk., Elmer B; Sanofi, Genomic Medicine Unit, Waltham, MA 02451, USA. Electronic address: bradford.elmer@sanofi.com., Noseworthy K; Biological Sciences, Hurvitz Brain Sciences Research Program, Sunnybrook Research Institute, Toronto, ON M4N 3M5, Canada; Department of Laboratory Medicine and Pathobiology, Temerty Faculty of Medicine, University of Toronto, Toronto, ON M5S 3K3, Canada., Ardinger J; Sanofi, Genomic Medicine Unit, Waltham, MA 02451, USA., Wu K; Biological Sciences, Hurvitz Brain Sciences Research Program, Sunnybrook Research Institute, Toronto, ON M4N 3M5, Canada; Department of Laboratory Medicine and Pathobiology, Temerty Faculty of Medicine, University of Toronto, Toronto, ON M5S 3K3, Canada., Wischhof E; Sanofi, Genomic Medicine Unit, Waltham, MA 02451, USA., Vecchio LM; Biological Sciences, Hurvitz Brain Sciences Research Program, Sunnybrook Research Institute, Toronto, ON M4N 3M5, Canada; Department of Laboratory Medicine and Pathobiology, Temerty Faculty of Medicine, University of Toronto, Toronto, ON M5S 3K3, Canada., Mahendran TA; Sanofi, Genomic Medicine Unit, Waltham, MA 02451, USA., Dibia CL; Biological Sciences, Hurvitz Brain Sciences Research Program, Sunnybrook Research Institute, Toronto, ON M4N 3M5, Canada; Department of Laboratory Medicine and Pathobiology, Temerty Faculty of Medicine, University of Toronto, Toronto, ON M5S 3K3, Canada., Koch H; Sanofi, Evidence Generation and Decision Sciences, Cambridge, MA 02141, USA., Chou SC; Sanofi, Genomic Medicine Unit, Waltham, MA 02451, USA., Sivadas S; Biological Sciences, Hurvitz Brain Sciences Research Program, Sunnybrook Research Institute, Toronto, ON M4N 3M5, Canada., Bu J; Sanofi, Genomic Medicine Unit, Waltham, MA 02451, USA., Wu SK; Physical Sciences, Sunnybrook Research Institute, Toronto, ON M4N 3M5, Canada; Department of Medical Biophysics, Temerty Faculty of Medicine, University of Toronto, Toronto, ON M5G 2C4, Canada., McMahon D; Physical Sciences, Sunnybrook Research Institute, Toronto, ON M4N 3M5, Canada., Mikloska K; Physical Sciences, Sunnybrook Research Institute, Toronto, ON M4N 3M5, Canada; Department of Medical Biophysics, Temerty Faculty of Medicine, University of Toronto, Toronto, ON M5G 2C4, Canada., White M; Biological Sciences, Hurvitz Brain Sciences Research Program, Sunnybrook Research Institute, Toronto, ON M4N 3M5, Canada; Department of Laboratory Medicine and Pathobiology, Temerty Faculty of Medicine, University of Toronto, Toronto, ON M5S 3K3, Canada., Ramachandran S; Sanofi, Genomic Medicine Unit, Waltham, MA 02451, USA., Hynynen K; Physical Sciences, Sunnybrook Research Institute, Toronto, ON M4N 3M5, Canada; Department of Medical Biophysics, Temerty Faculty of Medicine, University of Toronto, Toronto, ON M5G 2C4, Canada., Mueller C; Sanofi, Genomic Medicine Unit, Waltham, MA 02451, USA., Aubert I; Biological Sciences, Hurvitz Brain Sciences Research Program, Sunnybrook Research Institute, Toronto, ON M4N 3M5, Canada; Department of Laboratory Medicine and Pathobiology, Temerty Faculty of Medicine, University of Toronto, Toronto, ON M5S 3K3, Canada. Electronic address: isabelle.aubert@sri.utoronto.ca.
Source: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2026 May 06; Vol. 34 (5), pp. 2863-2878. Date of Electronic Publication: 2026 Feb 13.
Publication Type: Journal Article
Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 100890581 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1525-0024 (Electronic) Linking ISSN: 15250016 NLM ISO Abbreviation: Mol Ther Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: Focused ultrasound expands intra-CSF AAV delivery to deep brain regions in rats and non-human primates.
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  Data: <searchLink fieldCode="AU" term="%22Kofoed+RH%22">Kofoed RH</searchLink>; Department of Neurosurgery, Department of Clinical Medicine, Aarhus University, 8200 Aarhus N, Denmark; Center for Experimental Neuroscience - CENSE, Department of Neurosurgery, Aarhus University Hospital, 8200 Aarhus N, Denmark; Biological Sciences, Hurvitz Brain Sciences Research Program, Sunnybrook Research Institute, Toronto, ON M4N 3M5, Canada. Electronic address: rhko@clin.au.dk.<br /><searchLink fieldCode="AU" term="%22Elmer+B%22">Elmer B</searchLink>; Sanofi, Genomic Medicine Unit, Waltham, MA 02451, USA. Electronic address: bradford.elmer@sanofi.com.<br /><searchLink fieldCode="AU" term="%22Noseworthy+K%22">Noseworthy K</searchLink>; Biological Sciences, Hurvitz Brain Sciences Research Program, Sunnybrook Research Institute, Toronto, ON M4N 3M5, Canada; Department of Laboratory Medicine and Pathobiology, Temerty Faculty of Medicine, University of Toronto, Toronto, ON M5S 3K3, Canada.<br /><searchLink fieldCode="AU" term="%22Ardinger+J%22">Ardinger J</searchLink>; Sanofi, Genomic Medicine Unit, Waltham, MA 02451, USA.<br /><searchLink fieldCode="AU" term="%22Wu+K%22">Wu K</searchLink>; Biological Sciences, Hurvitz Brain Sciences Research Program, Sunnybrook Research Institute, Toronto, ON M4N 3M5, Canada; Department of Laboratory Medicine and Pathobiology, Temerty Faculty of Medicine, University of Toronto, Toronto, ON M5S 3K3, Canada.<br /><searchLink fieldCode="AU" term="%22Wischhof+E%22">Wischhof E</searchLink>; Sanofi, Genomic Medicine Unit, Waltham, MA 02451, USA.<br /><searchLink fieldCode="AU" term="%22Vecchio+LM%22">Vecchio LM</searchLink>; Biological Sciences, Hurvitz Brain Sciences Research Program, Sunnybrook Research Institute, Toronto, ON M4N 3M5, Canada; Department of Laboratory Medicine and Pathobiology, Temerty Faculty of Medicine, University of Toronto, Toronto, ON M5S 3K3, Canada.<br /><searchLink fieldCode="AU" term="%22Mahendran+TA%22">Mahendran TA</searchLink>; Sanofi, Genomic Medicine Unit, Waltham, MA 02451, USA.<br /><searchLink fieldCode="AU" term="%22Dibia+CL%22">Dibia CL</searchLink>; Biological Sciences, Hurvitz Brain Sciences Research Program, Sunnybrook Research Institute, Toronto, ON M4N 3M5, Canada; Department of Laboratory Medicine and Pathobiology, Temerty Faculty of Medicine, University of Toronto, Toronto, ON M5S 3K3, Canada.<br /><searchLink fieldCode="AU" term="%22Koch+H%22">Koch H</searchLink>; Sanofi, Evidence Generation and Decision Sciences, Cambridge, MA 02141, USA.<br /><searchLink fieldCode="AU" term="%22Chou+SC%22">Chou SC</searchLink>; Sanofi, Genomic Medicine Unit, Waltham, MA 02451, USA.<br /><searchLink fieldCode="AU" term="%22Sivadas+S%22">Sivadas S</searchLink>; Biological Sciences, Hurvitz Brain Sciences Research Program, Sunnybrook Research Institute, Toronto, ON M4N 3M5, Canada.<br /><searchLink fieldCode="AU" term="%22Bu+J%22">Bu J</searchLink>; Sanofi, Genomic Medicine Unit, Waltham, MA 02451, USA.<br /><searchLink fieldCode="AU" term="%22Wu+SK%22">Wu SK</searchLink>; Physical Sciences, Sunnybrook Research Institute, Toronto, ON M4N 3M5, Canada; Department of Medical Biophysics, Temerty Faculty of Medicine, University of Toronto, Toronto, ON M5G 2C4, Canada.<br /><searchLink fieldCode="AU" term="%22McMahon+D%22">McMahon D</searchLink>; Physical Sciences, Sunnybrook Research Institute, Toronto, ON M4N 3M5, Canada.<br /><searchLink fieldCode="AU" term="%22Mikloska+K%22">Mikloska K</searchLink>; Physical Sciences, Sunnybrook Research Institute, Toronto, ON M4N 3M5, Canada; Department of Medical Biophysics, Temerty Faculty of Medicine, University of Toronto, Toronto, ON M5G 2C4, Canada.<br /><searchLink fieldCode="AU" term="%22White+M%22">White M</searchLink>; Biological Sciences, Hurvitz Brain Sciences Research Program, Sunnybrook Research Institute, Toronto, ON M4N 3M5, Canada; Department of Laboratory Medicine and Pathobiology, Temerty Faculty of Medicine, University of Toronto, Toronto, ON M5S 3K3, Canada.<br /><searchLink fieldCode="AU" term="%22Ramachandran+S%22">Ramachandran S</searchLink>; Sanofi, Genomic Medicine Unit, Waltham, MA 02451, USA.<br /><searchLink fieldCode="AU" term="%22Hynynen+K%22">Hynynen K</searchLink>; Physical Sciences, Sunnybrook Research Institute, Toronto, ON M4N 3M5, Canada; Department of Medical Biophysics, Temerty Faculty of Medicine, University of Toronto, Toronto, ON M5G 2C4, Canada.<br /><searchLink fieldCode="AU" term="%22Mueller+C%22">Mueller C</searchLink>; Sanofi, Genomic Medicine Unit, Waltham, MA 02451, USA.<br /><searchLink fieldCode="AU" term="%22Aubert+I%22">Aubert I</searchLink>; Biological Sciences, Hurvitz Brain Sciences Research Program, Sunnybrook Research Institute, Toronto, ON M4N 3M5, Canada; Department of Laboratory Medicine and Pathobiology, Temerty Faculty of Medicine, University of Toronto, Toronto, ON M5S 3K3, Canada. Electronic address: isabelle.aubert@sri.utoronto.ca.
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