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. |
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| 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 |
| Database: | MEDLINE Ultimate |
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