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Treatment of a severe vascular disease using a bespoke CRISPR-Cas9 base editor in mice.

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Title: Treatment of a severe vascular disease using a bespoke CRISPR-Cas9 base editor in mice.
Authors: Alves CRR; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.; Department of Neurology, Harvard Medical School, Boston, MA, USA., Das S; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA., Krishnan V; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA., Ha LL; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA., Fox LR; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA., Stutzman HE; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA., Shamber CE; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA., Kalailingam P; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA., McCarthy S; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA., Lino Cardenas CL; Division of Cardiology, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Cardiovascular Research Center, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA., Fong CE; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA., Imai T; Neurovascular Research Unit, Department of Radiology, Massachusetts General Hospital, Harvard Medical School, Charlestown, MA, USA., Mitra S; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA., Yun S; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA., Wood RK; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA., Benning FMC; Department of Molecular Biology, Massachusetts General Hospital, Boston, MA, USA.; Department of Genetics, Harvard Medical School, Boston, MA, USA., Roh K; Division of Cardiology, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Cardiovascular Research Center, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA., Lawton J; Division of Cardiology, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Cardiovascular Research Center, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA., Kim N; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Harvard Medical School, Boston, MA, USA., Silverstein RA; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.; Biological and Biomedical Sciences Program, Harvard University, Boston, MA, USA., Ferreira da Silva J; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Harvard Medical School, Boston, MA, USA., de la Cruz D; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.; Department of Neurology, Harvard Medical School, Boston, MA, USA., Richa R; Division of Cardiology, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Cardiovascular Research Center, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA., Xie J; Department of Genetics and Cellular Medicine, UMass Chan Medical School, Worcester, MA, USA., Gray-Edwards HL; Department of Genetics and Cellular Medicine, UMass Chan Medical School, Worcester, MA, USA., Malhotra R; Division of Cardiology, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Cardiovascular Research Center, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA., Chung DY; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.; Department of Neurology, Harvard Medical School, Boston, MA, USA.; Neurovascular Research Unit, Department of Radiology, Massachusetts General Hospital, Harvard Medical School, Charlestown, MA, USA.; Division of Neurocritical Care, Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA., Chao LH; Department of Molecular Biology, Massachusetts General Hospital, Boston, MA, USA.; Department of Genetics, Harvard Medical School, Boston, MA, USA., Tsai SQ; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA., Maguire CA; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.; Department of Neurology, Harvard Medical School, Boston, MA, USA., Lindsay ME; Division of Cardiology, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA. Lindsay.Mark@mgh.harvard.edu.; Cardiovascular Research Center, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA. Lindsay.Mark@mgh.harvard.edu., Kleinstiver BP; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA. bkleinstiver@mgh.harvard.edu.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA. bkleinstiver@mgh.harvard.edu.; Department of Pathology, Harvard Medical School, Boston, MA, USA. bkleinstiver@mgh.harvard.edu., Musolino PL; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA. pmusolino@mgh.harvard.edu.; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA. pmusolino@mgh.harvard.edu.; Department of Neurology, Harvard Medical School, Boston, MA, USA. pmusolino@mgh.harvard.edu.; Division of Neurocritical Care, Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA. pmusolino@mgh.harvard.edu.
Source: Nature biomedical engineering [Nat Biomed Eng] 2026 May; Vol. 10 (5), pp. 952-967. Date of Electronic Publication: 2025 Sep 11.
Publication Type: Journal Article
Journal Info: Publisher: Springer Nature Country of Publication: England NLM ID: 101696896 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2157-846X (Electronic) Linking ISSN: 2157846X NLM ISO Abbreviation: Nat Biomed Eng Subsets: MEDLINE
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  Data: Treatment of a severe vascular disease using a bespoke CRISPR-Cas9 base editor in mice.
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  Data: <searchLink fieldCode="AU" term="%22Alves+CRR%22">Alves CRR</searchLink>; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.; Department of Neurology, Harvard Medical School, Boston, MA, USA.<br /><searchLink fieldCode="AU" term="%22Das+S%22">Das S</searchLink>; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.<br /><searchLink fieldCode="AU" term="%22Krishnan+V%22">Krishnan V</searchLink>; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.<br /><searchLink fieldCode="AU" term="%22Ha+LL%22">Ha LL</searchLink>; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.<br /><searchLink fieldCode="AU" term="%22Fox+LR%22">Fox LR</searchLink>; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.<br /><searchLink fieldCode="AU" term="%22Stutzman+HE%22">Stutzman HE</searchLink>; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.<br /><searchLink fieldCode="AU" term="%22Shamber+CE%22">Shamber CE</searchLink>; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.<br /><searchLink fieldCode="AU" term="%22Kalailingam+P%22">Kalailingam P</searchLink>; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.<br /><searchLink fieldCode="AU" term="%22McCarthy+S%22">McCarthy S</searchLink>; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.<br /><searchLink fieldCode="AU" term="%22Lino+Cardenas+CL%22">Lino Cardenas CL</searchLink>; Division of Cardiology, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Cardiovascular Research Center, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.<br /><searchLink fieldCode="AU" term="%22Fong+CE%22">Fong CE</searchLink>; 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Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.; Biological and Biomedical Sciences Program, Harvard University, Boston, MA, USA.<br /><searchLink fieldCode="AU" term="%22Ferreira+da+Silva+J%22">Ferreira da Silva J</searchLink>; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Harvard Medical School, Boston, MA, USA.<br /><searchLink fieldCode="AU" term="%22de+la+Cruz+D%22">de la Cruz D</searchLink>; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.; Department of Neurology, Harvard Medical School, Boston, MA, USA.<br /><searchLink fieldCode="AU" term="%22Richa+R%22">Richa R</searchLink>; Division of Cardiology, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Cardiovascular Research Center, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.<br /><searchLink fieldCode="AU" term="%22Xie+J%22">Xie J</searchLink>; Department of Genetics and Cellular Medicine, UMass Chan Medical School, Worcester, MA, USA.<br /><searchLink fieldCode="AU" term="%22Gray-Edwards+HL%22">Gray-Edwards HL</searchLink>; Department of Genetics and Cellular Medicine, UMass Chan Medical School, Worcester, MA, USA.<br /><searchLink fieldCode="AU" term="%22Malhotra+R%22">Malhotra R</searchLink>; Division of Cardiology, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Cardiovascular Research Center, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.<br /><searchLink fieldCode="AU" term="%22Chung+DY%22">Chung DY</searchLink>; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.; Department of Neurology, Harvard Medical School, Boston, MA, USA.; Neurovascular Research Unit, Department of Radiology, Massachusetts General Hospital, Harvard Medical School, Charlestown, MA, USA.; Division of Neurocritical Care, Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.<br /><searchLink fieldCode="AU" term="%22Chao+LH%22">Chao LH</searchLink>; Department of Molecular Biology, Massachusetts General Hospital, Boston, MA, USA.; Department of Genetics, Harvard Medical School, Boston, MA, USA.<br /><searchLink fieldCode="AU" term="%22Tsai+SQ%22">Tsai SQ</searchLink>; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.<br /><searchLink fieldCode="AU" term="%22Maguire+CA%22">Maguire CA</searchLink>; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.; Department of Neurology, Harvard Medical School, Boston, MA, USA.<br /><searchLink fieldCode="AU" term="%22Lindsay+ME%22">Lindsay ME</searchLink>; Division of Cardiology, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA. 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  Data: <searchLink fieldCode="JN" term="%22101696896%22">Nature biomedical engineering</searchLink> [Nat Biomed Eng] 2026 May; Vol. 10 (5), pp. 952-967. <i>Date of Electronic Publication: </i>2025 Sep 11.
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