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Paper Details

CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia.
N Engl J Med
748
2021
-Thalassemia, BCL11A, BCL11A erythroid-specific enhancer, CD34+, CD34+ hematopoietic stem and progenitor cells, CLIMB SCD-121, CLIMB THAL-111, CRISPR, Cas9, SCD, Sickle Cell Disease, TDT, Transfusion-dependent -thalassemia, autologous CD34+ cells, enhancer, erythroid cells, fetal hemoglobin, monogenic diseases, patient, patients, sickle cell disease, vaso-occlusive episodes
Author NameAffiliation
David AltshulerFrom the Sarah Cannon Center for Blood Cancer at the Children's Hospital at TriStar Centennial, and St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee Vertex Pharmaceuticals (D.A., A.Y.) and Boston University School of Medicine (M.H.S.), S. Soni) - both in Massachusetts the University of Milan, University of Rome, Rome (F.L.) the University of Regensburg, and Children's University Hospital, University of Tubingen, Tubingen (R.H.) - both in Germany Imperial College Healthcare NHS Trust, St. Mary's Hospital, London (J. de la Fuente) Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.G.) the University of Athens, Athens (A. Kattamis) BC Children's Hospital, University of British Columbia, and the Hospital for Sick Children-University of Toronto, Toronto (D.W.) - both in Canada Columbia University (M.Y.M.) and the Joan and Sanford I. Weill Medical College of Cornell University (S. Sheth), New York Necker-Enfants Malades Hospital, Assistance Publique-Hopitaux de Paris, University of Paris, Paris (M.M.) and the University of Illinois at Chicago
David AltshulerFrom the Sarah Cannon Center for Blood Cancer at the Children's Hospital at TriStar Centennial, and St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee Vertex Pharmaceuticals (D.A., A.Y.) and Boston University School of Medicine (M.H.S.), S. Soni) - both in Massachusetts the University of Milan, University of Rome, Rome (F.L.) the University of Regensburg, and Children's University Hospital, University of Tubingen, Tubingen (R.H.) - both in Germany Imperial College Healthcare NHS Trust, St. Mary's Hospital, London (J. de la Fuente) Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.G.) the University of Athens, Athens (A. Kattamis) BC Children's Hospital, University of British Columbia, and the Hospital for Sick Children-University of Toronto, Toronto (D.W.) - both in Canada Columbia University (M.Y.M.) and the Joan and Sanford I. Weill Medical College of Cornell University (S. Sheth), New York Necker-Enfants Malades Hospital, Assistance Publique-Hopitaux de Paris, University of Paris, Paris (M.M.) and the University of Illinois at Chicago
Stephan A GruppFrom the Sarah Cannon Center for Blood Cancer at the Children's Hospital at TriStar Centennial, and St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee Vertex Pharmaceuticals (D.A., A.Y.) and Boston University School of Medicine (M.H.S.), S. Soni) - both in Massachusetts the University of Milan, University of Rome, Rome (F.L.) the University of Regensburg, and Children's University Hospital, University of Tubingen, Tubingen (R.H.) - both in Germany Imperial College Healthcare NHS Trust, St. Mary's Hospital, London (J. de la Fuente) Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.G.) the University of Athens, Athens (A. Kattamis) BC Children's Hospital, University of British Columbia, and the Hospital for Sick Children-University of Toronto, Toronto (D.W.) - both in Canada Columbia University (M.Y.M.) and the Joan and Sanford I. Weill Medical College of Cornell University (S. Sheth), New York Necker-Enfants Malades Hospital, Assistance Publique-Hopitaux de Paris, University of Paris, Paris (M.M.) and the University of Illinois at Chicago
Angela YenFrom the Sarah Cannon Center for Blood Cancer at the Children's Hospital at TriStar Centennial, and St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee Vertex Pharmaceuticals (D.A., A.Y.) and Boston University School of Medicine (M.H.S.), S. Soni) - both in Massachusetts the University of Milan, University of Rome, Rome (F.L.) the University of Regensburg, and Children's University Hospital, University of Tubingen, Tubingen (R.H.) - both in Germany Imperial College Healthcare NHS Trust, St. Mary's Hospital, London (J. de la Fuente) Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.G.) the University of Athens, Athens (A. Kattamis) BC Children's Hospital, University of British Columbia, and the Hospital for Sick Children-University of Toronto, Toronto (D.W.) - both in Canada Columbia University (M.Y.M.) and the Joan and Sanford I. Weill Medical College of Cornell University (S. Sheth), New York Necker-Enfants Malades Hospital, Assistance Publique-Hopitaux de Paris, University of Paris, Paris (M.M.) and the University of Illinois at Chicago
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Datasets

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ClinicalTrials.govClinicalTrials.gov, a registry and results database of of privately and publicly funded clinical studies conducted around the world, is operated by the U.S. National Library of Medicine (NLM). Structured study information is provided, voluntarily or to meet legal and policy requirements, by the sponsor or investigator responsible for the research. NLM does not verify the scientific validity or relevance of the submitted information beyond a limited quality control review for apparent errors, deficiencies, or inconsistencies.Link
ClinicalTrials.govClinicalTrials.gov, a registry and results database of of privately and publicly funded clinical studies conducted around the world, is operated by the U.S. National Library of Medicine (NLM). Structured study information is provided, voluntarily or to meet legal and policy requirements, by the sponsor or investigator responsible for the research. NLM does not verify the scientific validity or relevance of the submitted information beyond a limited quality control review for apparent errors, deficiencies, or inconsistencies.Link
ClinicalTrials.govClinicalTrials.gov, a registry and results database of of privately and publicly funded clinical studies conducted around the world, is operated by the U.S. National Library of Medicine (NLM). Structured study information is provided, voluntarily or to meet legal and policy requirements, by the sponsor or investigator responsible for the research. NLM does not verify the scientific validity or relevance of the submitted information beyond a limited quality control review for apparent errors, deficiencies, or inconsistencies.Link
ClinicalTrials.govClinicalTrials.gov, a registry and results database of of privately and publicly funded clinical studies conducted around the world, is operated by the U.S. National Library of Medicine (NLM). Structured study information is provided, voluntarily or to meet legal and policy requirements, by the sponsor or investigator responsible for the research. NLM does not verify the scientific validity or relevance of the submitted information beyond a limited quality control review for apparent errors, deficiencies, or inconsistencies.Link
ClinicalTrials.govClinicalTrials.gov, a registry and results database of of privately and publicly funded clinical studies conducted around the world, is operated by the U.S. National Library of Medicine (NLM). Structured study information is provided, voluntarily or to meet legal and policy requirements, by the sponsor or investigator responsible for the research. NLM does not verify the scientific validity or relevance of the submitted information beyond a limited quality control review for apparent errors, deficiencies, or inconsistencies.Link
ClinicalTrials.govClinicalTrials.gov, a registry and results database of of privately and publicly funded clinical studies conducted around the world, is operated by the U.S. National Library of Medicine (NLM). Structured study information is provided, voluntarily or to meet legal and policy requirements, by the sponsor or investigator responsible for the research. NLM does not verify the scientific validity or relevance of the submitted information beyond a limited quality control review for apparent errors, deficiencies, or inconsistencies.Link
ClinicalTrials.govClinicalTrials.gov, a registry and results database of of privately and publicly funded clinical studies conducted around the world, is operated by the U.S. National Library of Medicine (NLM). Structured study information is provided, voluntarily or to meet legal and policy requirements, by the sponsor or investigator responsible for the research. NLM does not verify the scientific validity or relevance of the submitted information beyond a limited quality control review for apparent errors, deficiencies, or inconsistencies.Link
ClinicalTrials.govClinicalTrials.gov, a registry and results database of of privately and publicly funded clinical studies conducted around the world, is operated by the U.S. National Library of Medicine (NLM). Structured study information is provided, voluntarily or to meet legal and policy requirements, by the sponsor or investigator responsible for the research. NLM does not verify the scientific validity or relevance of the submitted information beyond a limited quality control review for apparent errors, deficiencies, or inconsistencies.Link
ClinicalTrials.govClinicalTrials.gov, a registry and results database of of privately and publicly funded clinical studies conducted around the world, is operated by the U.S. National Library of Medicine (NLM). Structured study information is provided, voluntarily or to meet legal and policy requirements, by the sponsor or investigator responsible for the research. NLM does not verify the scientific validity or relevance of the submitted information beyond a limited quality control review for apparent errors, deficiencies, or inconsistencies.Link
ClinicalTrials.govClinicalTrials.gov, a registry and results database of of privately and publicly funded clinical studies conducted around the world, is operated by the U.S. National Library of Medicine (NLM). Structured study information is provided, voluntarily or to meet legal and policy requirements, by the sponsor or investigator responsible for the research. NLM does not verify the scientific validity or relevance of the submitted information beyond a limited quality control review for apparent errors, deficiencies, or inconsistencies.Link
ClinicalTrials.govClinicalTrials.gov, a registry and results database of of privately and publicly funded clinical studies conducted around the world, is operated by the U.S. National Library of Medicine (NLM). Structured study information is provided, voluntarily or to meet legal and policy requirements, by the sponsor or investigator responsible for the research. NLM does not verify the scientific validity or relevance of the submitted information beyond a limited quality control review for apparent errors, deficiencies, or inconsistencies.Link
ClinicalTrials.govClinicalTrials.gov, a registry and results database of of privately and publicly funded clinical studies conducted around the world, is operated by the U.S. National Library of Medicine (NLM). Structured study information is provided, voluntarily or to meet legal and policy requirements, by the sponsor or investigator responsible for the research. NLM does not verify the scientific validity or relevance of the submitted information beyond a limited quality control review for apparent errors, deficiencies, or inconsistencies.Link
ClinicalTrials.govClinicalTrials.gov, a registry and results database of of privately and publicly funded clinical studies conducted around the world, is operated by the U.S. National Library of Medicine (NLM). Structured study information is provided, voluntarily or to meet legal and policy requirements, by the sponsor or investigator responsible for the research. NLM does not verify the scientific validity or relevance of the submitted information beyond a limited quality control review for apparent errors, deficiencies, or inconsistencies.Link
ClinicalTrials.govClinicalTrials.gov, a registry and results database of of privately and publicly funded clinical studies conducted around the world, is operated by the U.S. National Library of Medicine (NLM). Structured study information is provided, voluntarily or to meet legal and policy requirements, by the sponsor or investigator responsible for the research. NLM does not verify the scientific validity or relevance of the submitted information beyond a limited quality control review for apparent errors, deficiencies, or inconsistencies.Link
ClinicalTrials.govClinicalTrials.gov, a registry and results database of of privately and publicly funded clinical studies conducted around the world, is operated by the U.S. National Library of Medicine (NLM). Structured study information is provided, voluntarily or to meet legal and policy requirements, by the sponsor or investigator responsible for the research. NLM does not verify the scientific validity or relevance of the submitted information beyond a limited quality control review for apparent errors, deficiencies, or inconsistencies.Link
Release 1.2