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Prime Editing for p47phox-Deficient Chronic Granulomatous Disease.

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Title: Prime Editing for p47phox-Deficient Chronic Granulomatous Disease.
Authors: Gori JL; Prime Medicine, Cambridge, MA., Haddad E; Department of Pediatrics, CHU Sainte-Justine Azrieli Research Center, Université de Montréal, Montreal.; Department of Microbiology, Immunology, and Infectious Diseases, CHU Sainte-Justine Azrieli Research Center, Université de Montréal, Montreal., Frangoul H; Sarah Cannon Research Institute, Children's Hospital at TriStar Centennial, Nashville., Kohn DB; Department of Pediatrics, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles., Morris EC; National Institute for Health and Care Research, University College London Hospitals Biomedical Research Centre, London., Martin BN; Prime Medicine, Cambridge, MA., Deary BA; Prime Medicine, Cambridge, MA., Nickerson M; Prime Medicine, Cambridge, MA., Scholz RL; Prime Medicine, Cambridge, MA., Fernandez I; Department of Pediatrics, CHU Sainte-Justine Azrieli Research Center, Université de Montréal, Montreal.; Department of Microbiology, Immunology, and Infectious Diseases, CHU Sainte-Justine Azrieli Research Center, Université de Montréal, Montreal., Leveille K; Department of Pediatrics, CHU Sainte-Justine Azrieli Research Center, Université de Montréal, Montreal.; Department of Microbiology, Immunology, and Infectious Diseases, CHU Sainte-Justine Azrieli Research Center, Université de Montréal, Montreal., De Ravin SS; Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD., Kang EM; Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD., Pierzynski M; Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD., Estwick T; Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD., Littel P; Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD., Kuhns DB; Leidos Biomedical Research, Frederick National Laboratory for Cancer Research, Frederick, MD., Long Priel DA; Leidos Biomedical Research, Frederick National Laboratory for Cancer Research, Frederick, MD., Teira P; Department of Pediatrics, CHU Sainte-Justine Azrieli Research Center, Université de Montréal, Montreal., Turvey S; Department of Pediatrics, British Columbia Children's Hospital, University of British Columbia, Vancouver, Canada., Arnold J; Department of Pediatrics, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles., Evans MD; Sarah Cannon Research Institute, Children's Hospital at TriStar Centennial, Nashville., McManus M; Sarah Cannon Research Institute, Children's Hospital at TriStar Centennial, Nashville., Carpenter B; Department of Hematology, University College London Hospitals NHS Foundation Trust, London., Waterman DP; Prime Medicine, Cambridge, MA., Anzalone AV; Prime Medicine, Cambridge, MA., Petrusich A; Prime Medicine, Cambridge, MA., Osuna CE; Prime Medicine, Cambridge, MA., O'Malley TT; Prime Medicine, Cambridge, MA., Stewart-Ornstein J; Prime Medicine, Cambridge, MA., Heath JM; Prime Medicine, Cambridge, MA., Nehilla BJ; Prime Medicine, Cambridge, MA., Asmal M; Prime Medicine, Cambridge, MA., Malech HL; Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD.
Source: The New England journal of medicine [N Engl J Med] 2026 Mar 26; Vol. 394 (12), pp. 1195-1203. Date of Electronic Publication: 2025 Dec 07.
Publication Type: Clinical Trial, Phase I; Clinical Trial, Phase II; Journal Article; Multicenter Study
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
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  Data: Prime Editing for p47<superscript>phox</superscript>-Deficient Chronic Granulomatous Disease.
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  Data: <searchLink fieldCode="AU" term="%22Gori+JL%22">Gori JL</searchLink>; Prime Medicine, Cambridge, MA.<br /><searchLink fieldCode="AU" term="%22Haddad+E%22">Haddad E</searchLink>; Department of Pediatrics, CHU Sainte-Justine Azrieli Research Center, Université de Montréal, Montreal.; Department of Microbiology, Immunology, and Infectious Diseases, CHU Sainte-Justine Azrieli Research Center, Université de Montréal, Montreal.<br /><searchLink fieldCode="AU" term="%22Frangoul+H%22">Frangoul H</searchLink>; Sarah Cannon Research Institute, Children's Hospital at TriStar Centennial, Nashville.<br /><searchLink fieldCode="AU" term="%22Kohn+DB%22">Kohn DB</searchLink>; Department of Pediatrics, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles.<br /><searchLink fieldCode="AU" term="%22Morris+EC%22">Morris EC</searchLink>; National Institute for Health and Care Research, University College London Hospitals Biomedical Research Centre, London.<br /><searchLink fieldCode="AU" term="%22Martin+BN%22">Martin BN</searchLink>; Prime Medicine, Cambridge, MA.<br /><searchLink fieldCode="AU" term="%22Deary+BA%22">Deary BA</searchLink>; Prime Medicine, Cambridge, MA.<br /><searchLink fieldCode="AU" term="%22Nickerson+M%22">Nickerson M</searchLink>; Prime Medicine, Cambridge, MA.<br /><searchLink fieldCode="AU" term="%22Scholz+RL%22">Scholz RL</searchLink>; Prime Medicine, Cambridge, MA.<br /><searchLink fieldCode="AU" term="%22Fernandez+I%22">Fernandez I</searchLink>; Department of Pediatrics, CHU Sainte-Justine Azrieli Research Center, Université de Montréal, Montreal.; Department of Microbiology, Immunology, and Infectious Diseases, CHU Sainte-Justine Azrieli Research Center, Université de Montréal, Montreal.<br /><searchLink fieldCode="AU" term="%22Leveille+K%22">Leveille K</searchLink>; Department of Pediatrics, CHU Sainte-Justine Azrieli Research Center, Université de Montréal, Montreal.; Department of Microbiology, Immunology, and Infectious Diseases, CHU Sainte-Justine Azrieli Research Center, Université de Montréal, Montreal.<br /><searchLink fieldCode="AU" term="%22De+Ravin+SS%22">De Ravin SS</searchLink>; Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD.<br /><searchLink fieldCode="AU" term="%22Kang+EM%22">Kang EM</searchLink>; Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD.<br /><searchLink fieldCode="AU" term="%22Pierzynski+M%22">Pierzynski M</searchLink>; Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD.<br /><searchLink fieldCode="AU" term="%22Estwick+T%22">Estwick T</searchLink>; Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD.<br /><searchLink fieldCode="AU" term="%22Littel+P%22">Littel P</searchLink>; Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD.<br /><searchLink fieldCode="AU" term="%22Kuhns+DB%22">Kuhns DB</searchLink>; Leidos Biomedical Research, Frederick National Laboratory for Cancer Research, Frederick, MD.<br /><searchLink fieldCode="AU" term="%22Long+Priel+DA%22">Long Priel DA</searchLink>; Leidos Biomedical Research, Frederick National Laboratory for Cancer Research, Frederick, MD.<br /><searchLink fieldCode="AU" term="%22Teira+P%22">Teira P</searchLink>; Department of Pediatrics, CHU Sainte-Justine Azrieli Research Center, Université de Montréal, Montreal.<br /><searchLink fieldCode="AU" term="%22Turvey+S%22">Turvey S</searchLink>; Department of Pediatrics, British Columbia Children's Hospital, University of British Columbia, Vancouver, Canada.<br /><searchLink fieldCode="AU" term="%22Arnold+J%22">Arnold J</searchLink>; Department of Pediatrics, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles.<br /><searchLink fieldCode="AU" term="%22Evans+MD%22">Evans MD</searchLink>; Sarah Cannon Research Institute, Children's Hospital at TriStar Centennial, Nashville.<br /><searchLink fieldCode="AU" term="%22McManus+M%22">McManus M</searchLink>; Sarah Cannon Research Institute, Children's Hospital at TriStar Centennial, Nashville.<br /><searchLink fieldCode="AU" term="%22Carpenter+B%22">Carpenter B</searchLink>; Department of Hematology, University College London Hospitals NHS Foundation Trust, London.<br /><searchLink fieldCode="AU" term="%22Waterman+DP%22">Waterman DP</searchLink>; Prime Medicine, Cambridge, MA.<br /><searchLink fieldCode="AU" term="%22Anzalone+AV%22">Anzalone AV</searchLink>; Prime Medicine, Cambridge, MA.<br /><searchLink fieldCode="AU" term="%22Petrusich+A%22">Petrusich A</searchLink>; Prime Medicine, Cambridge, MA.<br /><searchLink fieldCode="AU" term="%22Osuna+CE%22">Osuna CE</searchLink>; Prime Medicine, Cambridge, MA.<br /><searchLink fieldCode="AU" term="%22O'Malley+TT%22">O'Malley TT</searchLink>; Prime Medicine, Cambridge, MA.<br /><searchLink fieldCode="AU" term="%22Stewart-Ornstein+J%22">Stewart-Ornstein J</searchLink>; Prime Medicine, Cambridge, MA.<br /><searchLink fieldCode="AU" term="%22Heath+JM%22">Heath JM</searchLink>; Prime Medicine, Cambridge, MA.<br /><searchLink fieldCode="AU" term="%22Nehilla+BJ%22">Nehilla BJ</searchLink>; Prime Medicine, Cambridge, MA.<br /><searchLink fieldCode="AU" term="%22Asmal+M%22">Asmal M</searchLink>; Prime Medicine, Cambridge, MA.<br /><searchLink fieldCode="AU" term="%22Malech+HL%22">Malech HL</searchLink>; Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD.
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  Data: <searchLink fieldCode="JN" term="%220255562%22">The New England journal of medicine</searchLink> [N Engl J Med] 2026 Mar 26; Vol. 394 (12), pp. 1195-1203. <i>Date of Electronic Publication: </i>2025 Dec 07.
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