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Human Lentiviral Gene Therapy Restores the Cellular Phenotype of Autosomal Recessive Complete IFN-γR1 Deficiency.

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Title: Human Lentiviral Gene Therapy Restores the Cellular Phenotype of Autosomal Recessive Complete IFN-γR1 Deficiency.
Authors: Hahn K; Translational Hematology of Congenital Diseases, Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany.; REBIRTH Research Center for Translational and Regenerative Medicine, Hannover, Germany., Pollmann L; Translational Hematology of Congenital Diseases, Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany.; REBIRTH Research Center for Translational and Regenerative Medicine, Hannover, Germany., Nowak J; Translational Hematology of Congenital Diseases, Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany.; REBIRTH Research Center for Translational and Regenerative Medicine, Hannover, Germany., Nguyen AHH; Translational Hematology of Congenital Diseases, Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany.; REBIRTH Research Center for Translational and Regenerative Medicine, Hannover, Germany., Haake K; Translational Hematology of Congenital Diseases, Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany.; REBIRTH Research Center for Translational and Regenerative Medicine, Hannover, Germany., Neehus AL; Translational Hematology of Congenital Diseases, Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany.; Laboratory of Human Genetics of Infectious Diseases, Necker Branch, INSERM U1163, Necker Hospital for Sick Children, Paris, France.; Imagine Institute, Paris University, Paris, France., Waqas SFH; Biomarkers for Infectious Diseases, TWINCORE, Centre for Experimental and Clinical Infection Research, Hannover, Germany., Pessler F; Biomarkers for Infectious Diseases, TWINCORE, Centre for Experimental and Clinical Infection Research, Hannover, Germany.; Helmholtz Centre for Infection Research, Brunswick, Germany., Baumann U; Department of Pediatric Pulmonology, Allergy and Neonatology, Hannover Medical School, Hannover, Germany., Hetzel M; Translational Hematology of Congenital Diseases, Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany.; REBIRTH Research Center for Translational and Regenerative Medicine, Hannover, Germany., Casanova JL; Laboratory of Human Genetics of Infectious Diseases, Necker Branch, INSERM U1163, Necker Hospital for Sick Children, Paris, France.; Imagine Institute, Paris University, Paris, France.; St. Giles Laboratory of Human Genetics of Infectious Diseases, Rockefeller Branch, The Rockefeller University, New York, NY, USA.; Howard Hughes Medical Institute, New York, NY, USA.; Pediatric Hematology and Immunology Unit, Necker Hospital for Sick Children, AP-HP, Paris, France., Schulz A; Department of Pediatrics and Adolescent Medicine, University Medical Center Ulm, Ulm, Germany., Bustamante J; Laboratory of Human Genetics of Infectious Diseases, Necker Branch, INSERM U1163, Necker Hospital for Sick Children, Paris, France.; Imagine Institute, Paris University, Paris, France.; St. Giles Laboratory of Human Genetics of Infectious Diseases, Rockefeller Branch, The Rockefeller University, New York, NY, USA.; Center for the Study of Primary Immunodeficiencies, Necker Hospital for Sick Children, AP-HP, Paris, France., Ackermann M; Translational Hematology of Congenital Diseases, Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany.; REBIRTH Research Center for Translational and Regenerative Medicine, Hannover, Germany., Lachmann N; Translational Hematology of Congenital Diseases, Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany.; REBIRTH Research Center for Translational and Regenerative Medicine, Hannover, Germany.
Source: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2020 Apr 11; Vol. 17, pp. 785-795. Date of Electronic Publication: 2020 Apr 11 (Print Publication: 2020).
Publication Type: Journal Article
Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 101624857 Publication Model: eCollection Cited Medium: Print ISSN: 2329-0501 (Print) Linking ISSN: 23290501 NLM ISO Abbreviation: Mol Ther Methods Clin Dev Subsets: PubMed not MEDLINE
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  Data: Human Lentiviral Gene Therapy Restores the Cellular Phenotype of Autosomal Recessive Complete IFN-γR1 Deficiency.
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  Data: <searchLink fieldCode="AU" term="%22Hahn+K%22">Hahn K</searchLink>; Translational Hematology of Congenital Diseases, Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany.; REBIRTH Research Center for Translational and Regenerative Medicine, Hannover, Germany.<br /><searchLink fieldCode="AU" term="%22Pollmann+L%22">Pollmann L</searchLink>; Translational Hematology of Congenital Diseases, Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany.; REBIRTH Research Center for Translational and Regenerative Medicine, Hannover, Germany.<br /><searchLink fieldCode="AU" term="%22Nowak+J%22">Nowak J</searchLink>; Translational Hematology of Congenital Diseases, Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany.; REBIRTH Research Center for Translational and Regenerative Medicine, Hannover, Germany.<br /><searchLink fieldCode="AU" term="%22Nguyen+AHH%22">Nguyen AHH</searchLink>; Translational Hematology of Congenital Diseases, Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany.; REBIRTH Research Center for Translational and Regenerative Medicine, Hannover, Germany.<br /><searchLink fieldCode="AU" term="%22Haake+K%22">Haake K</searchLink>; Translational Hematology of Congenital Diseases, Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany.; REBIRTH Research Center for Translational and Regenerative Medicine, Hannover, Germany.<br /><searchLink fieldCode="AU" term="%22Neehus+AL%22">Neehus AL</searchLink>; Translational Hematology of Congenital Diseases, Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany.; Laboratory of Human Genetics of Infectious Diseases, Necker Branch, INSERM U1163, Necker Hospital for Sick Children, Paris, France.; Imagine Institute, Paris University, Paris, France.<br /><searchLink fieldCode="AU" term="%22Waqas+SFH%22">Waqas SFH</searchLink>; Biomarkers for Infectious Diseases, TWINCORE, Centre for Experimental and Clinical Infection Research, Hannover, Germany.<br /><searchLink fieldCode="AU" term="%22Pessler+F%22">Pessler F</searchLink>; Biomarkers for Infectious Diseases, TWINCORE, Centre for Experimental and Clinical Infection Research, Hannover, Germany.; Helmholtz Centre for Infection Research, Brunswick, Germany.<br /><searchLink fieldCode="AU" term="%22Baumann+U%22">Baumann U</searchLink>; Department of Pediatric Pulmonology, Allergy and Neonatology, Hannover Medical School, Hannover, Germany.<br /><searchLink fieldCode="AU" term="%22Hetzel+M%22">Hetzel M</searchLink>; Translational Hematology of Congenital Diseases, Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany.; REBIRTH Research Center for Translational and Regenerative Medicine, Hannover, Germany.<br /><searchLink fieldCode="AU" term="%22Casanova+JL%22">Casanova JL</searchLink>; Laboratory of Human Genetics of Infectious Diseases, Necker Branch, INSERM U1163, Necker Hospital for Sick Children, Paris, France.; Imagine Institute, Paris University, Paris, France.; St. Giles Laboratory of Human Genetics of Infectious Diseases, Rockefeller Branch, The Rockefeller University, New York, NY, USA.; Howard Hughes Medical Institute, New York, NY, USA.; Pediatric Hematology and Immunology Unit, Necker Hospital for Sick Children, AP-HP, Paris, France.<br /><searchLink fieldCode="AU" term="%22Schulz+A%22">Schulz A</searchLink>; Department of Pediatrics and Adolescent Medicine, University Medical Center Ulm, Ulm, Germany.<br /><searchLink fieldCode="AU" term="%22Bustamante+J%22">Bustamante J</searchLink>; Laboratory of Human Genetics of Infectious Diseases, Necker Branch, INSERM U1163, Necker Hospital for Sick Children, Paris, France.; Imagine Institute, Paris University, Paris, France.; St. Giles Laboratory of Human Genetics of Infectious Diseases, Rockefeller Branch, The Rockefeller University, New York, NY, USA.; Center for the Study of Primary Immunodeficiencies, Necker Hospital for Sick Children, AP-HP, Paris, France.<br /><searchLink fieldCode="AU" term="%22Ackermann+M%22">Ackermann M</searchLink>; Translational Hematology of Congenital Diseases, Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany.; REBIRTH Research Center for Translational and Regenerative Medicine, Hannover, Germany.<br /><searchLink fieldCode="AU" term="%22Lachmann+N%22">Lachmann N</searchLink>; Translational Hematology of Congenital Diseases, Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany.; REBIRTH Research Center for Translational and Regenerative Medicine, Hannover, Germany.
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