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Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease.

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Title: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease.
Authors: Musunuru K; Children's Hospital of Philadelphia, Philadelphia.; Perelman School of Medicine at the University of Pennsylvania, Philadelphia., Grandinette SA; Perelman School of Medicine at the University of Pennsylvania, Philadelphia., Wang X; Perelman School of Medicine at the University of Pennsylvania, Philadelphia., Hudson TR; Innovative Genomics Institute, University of California, Berkeley, Berkeley., Briseno K; Innovative Genomics Institute, University of California, Berkeley, Berkeley., Berry AM; Perelman School of Medicine at the University of Pennsylvania, Philadelphia., Hacker JL; Perelman School of Medicine at the University of Pennsylvania, Philadelphia., Hsu A; Broad Institute of MIT and Harvard, Harvard University, Howard Hughes Medical Institute, Cambridge, MA., Silverstein RA; Massachusetts General Hospital-Harvard Medical School, Boston., Hille LT; Massachusetts General Hospital-Harvard Medical School, Boston., Ogul AN; Innovative Genomics Institute, University of California, Berkeley, Berkeley., Robinson-Garvin NA; Children's Hospital of Philadelphia, Philadelphia., Small JC; Children's Hospital of Philadelphia, Philadelphia., McCague S; Children's Hospital of Philadelphia, Philadelphia., Burke SM; Children's Hospital of Philadelphia, Philadelphia., Wright CM; Children's Hospital of Philadelphia, Philadelphia., Bick S; Children's Hospital of Philadelphia, Philadelphia., Indurthi V; Aldevron, Fargo, ND., Sharma S; Aldevron, Fargo, ND., Jepperson M; Aldevron, Fargo, ND., Vakulskas CA; Integrated DNA Technologies, Coralville, IA., Collingwood M; Integrated DNA Technologies, Coralville, IA., Keogh K; Integrated DNA Technologies, Coralville, IA., Jacobi A; Integrated DNA Technologies, Coralville, IA., Sturgeon M; Integrated DNA Technologies, Coralville, IA., Brommel C; Integrated DNA Technologies, Coralville, IA., Schmaljohn E; Integrated DNA Technologies, Coralville, IA., Kurgan G; Integrated DNA Technologies, Coralville, IA., Osborne T; Integrated DNA Technologies, Coralville, IA., Zhang H; Integrated DNA Technologies, Coralville, IA., Kinney K; Integrated DNA Technologies, Coralville, IA., Rettig G; Integrated DNA Technologies, Coralville, IA., Barbosa CJ; Acuitas Therapeutics, Vancouver, BC, Canada., Semple SC; Acuitas Therapeutics, Vancouver, BC, Canada., Tam YK; Acuitas Therapeutics, Vancouver, BC, Canada., Lutz C; Jackson Laboratory, Bar Harbor, ME., George LA; Children's Hospital of Philadelphia, Philadelphia.; Perelman School of Medicine at the University of Pennsylvania, Philadelphia., Kleinstiver BP; Massachusetts General Hospital-Harvard Medical School, Boston., Liu DR; Broad Institute of MIT and Harvard, Harvard University, Howard Hughes Medical Institute, Cambridge, MA., Ng K; Children's Hospital of Philadelphia, Philadelphia., Kassim SH; Danaher Corporation, Washington, DC., Giannikopoulos P; Innovative Genomics Institute, University of California, Berkeley, Berkeley.; University of California, San Francisco, San Francisco., Alameh MG; Children's Hospital of Philadelphia, Philadelphia.; Perelman School of Medicine at the University of Pennsylvania, Philadelphia., Urnov FD; Innovative Genomics Institute, University of California, Berkeley, Berkeley., Ahrens-Nicklas RC; Children's Hospital of Philadelphia, Philadelphia.; Perelman School of Medicine at the University of Pennsylvania, Philadelphia.
Source: The New England journal of medicine [N Engl J Med] 2025 Jun 12; Vol. 392 (22), pp. 2235-2243. Date of Electronic Publication: 2025 May 15.
Publication Type: Journal Article; Case Reports
Journal Info: Publisher: Massachusetts Medical Society Country of Publication: United States NLM ID: 0255562 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1533-4406 (Electronic) Linking ISSN: 00284793 NLM ISO Abbreviation: N Engl J Med Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease.
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Children's Hospital of Philadelphia, Philadelphia.<br /><searchLink fieldCode="AU" term="%22Kassim+SH%22">Kassim SH</searchLink>; Danaher Corporation, Washington, DC.<br /><searchLink fieldCode="AU" term="%22Giannikopoulos+P%22">Giannikopoulos P</searchLink>; Innovative Genomics Institute, University of California, Berkeley, Berkeley.; University of California, San Francisco, San Francisco.<br /><searchLink fieldCode="AU" term="%22Alameh+MG%22">Alameh MG</searchLink>; Children's Hospital of Philadelphia, Philadelphia.; Perelman School of Medicine at the University of Pennsylvania, Philadelphia.<br /><searchLink fieldCode="AU" term="%22Urnov+FD%22">Urnov FD</searchLink>; Innovative Genomics Institute, University of California, Berkeley, Berkeley.<br /><searchLink fieldCode="AU" term="%22Ahrens-Nicklas+RC%22">Ahrens-Nicklas RC</searchLink>; Children's Hospital of Philadelphia, Philadelphia.; Perelman School of Medicine at the University of Pennsylvania, Philadelphia.
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