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Autor:
Zabaleta N; Grousbeck Gene Therapy Center, Schepens Eye Research Institute, Mass Eye and Ear, Boston, MA, USA; Ocular Genomics Institute, Department of Ophthalmology, Harvard Medical School, Boston, MA, USA; The Broad Institute of Harvard and MIT, Cambridge, MA, USA; Harvard Stem Cell Institute, Harvard University, Cambridge, MA, USA., Dai W; Grousbeck Gene Therapy Center, Schepens Eye Research Institute, Mass Eye and Ear, Boston, MA, USA; Ocular Genomics Institute, Department of Ophthalmology, Harvard Medical School, Boston, MA, USA; The Broad Institute of Harvard and MIT, Cambridge, MA, USA; Harvard Stem Cell Institute, Harvard University, Cambridge, MA, USA., Bhatt U; Grousbeck Gene Therapy Center, Schepens Eye Research Institute, Mass Eye and Ear, Boston, MA, USA; Ocular Genomics Institute, Department of Ophthalmology, Harvard Medical School, Boston, MA, USA; The Broad Institute of Harvard and MIT, Cambridge, MA, USA; Harvard Stem Cell Institute, Harvard University, Cambridge, MA, USA., Hérate C; Center for Immunology of Viral, Auto-immune, Hematological and Bacterial diseases (IMVA-HB/IDMIT), Université Paris-Saclay, Inserm, CEA, Fontenay-aux-Roses, France., Maisonnasse P; Center for Immunology of Viral, Auto-immune, Hematological and Bacterial diseases (IMVA-HB/IDMIT), Université Paris-Saclay, Inserm, CEA, Fontenay-aux-Roses, France., Chichester JA; Gene Therapy Program, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Sanmiguel J; Grousbeck Gene Therapy Center, Schepens Eye Research Institute, Mass Eye and Ear, Boston, MA, USA; Ocular Genomics Institute, Department of Ophthalmology, Harvard Medical School, Boston, MA, USA; The Broad Institute of Harvard and MIT, Cambridge, MA, USA; Harvard Stem Cell Institute, Harvard University, Cambridge, MA, USA., Estelien R; Grousbeck Gene Therapy Center, Schepens Eye Research Institute, Mass Eye and Ear, Boston, MA, USA; Ocular Genomics Institute, Department of Ophthalmology, Harvard Medical School, Boston, MA, USA; The Broad Institute of Harvard and MIT, Cambridge, MA, USA; Harvard Stem Cell Institute, Harvard University, Cambridge, MA, USA., Michalson KT; Gene Therapy Program, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Diop C; Grousbeck Gene Therapy Center, Schepens Eye Research Institute, Mass Eye and Ear, Boston, MA, USA; Ocular Genomics Institute, Department of Ophthalmology, Harvard Medical School, Boston, MA, USA; The Broad Institute of Harvard and MIT, Cambridge, MA, USA; Harvard Stem Cell Institute, Harvard University, Cambridge, MA, USA., Maciorowski D; Grousbeck Gene Therapy Center, Schepens Eye Research Institute, Mass Eye and Ear, Boston, MA, USA; Ocular Genomics Institute, Department of Ophthalmology, Harvard Medical School, Boston, MA, USA; The Broad Institute of Harvard and MIT, Cambridge, MA, USA; Harvard Stem Cell Institute, Harvard University, Cambridge, MA, USA., Dereuddre-Bosquet N; Center for Immunology of Viral, Auto-immune, Hematological and Bacterial diseases (IMVA-HB/IDMIT), Université Paris-Saclay, Inserm, CEA, Fontenay-aux-Roses, France., Cavarelli M; Center for Immunology of Viral, Auto-immune, Hematological and Bacterial diseases (IMVA-HB/IDMIT), Université Paris-Saclay, Inserm, CEA, Fontenay-aux-Roses, France., Gallouët AS; Center for Immunology of Viral, Auto-immune, Hematological and Bacterial diseases (IMVA-HB/IDMIT), Université Paris-Saclay, Inserm, CEA, Fontenay-aux-Roses, France., Naninck T; Center for Immunology of Viral, Auto-immune, Hematological and Bacterial diseases (IMVA-HB/IDMIT), Université Paris-Saclay, Inserm, CEA, Fontenay-aux-Roses, France., Kahlaoui N; Center for Immunology of Viral, Auto-immune, Hematological and Bacterial diseases (IMVA-HB/IDMIT), Université Paris-Saclay, Inserm, CEA, Fontenay-aux-Roses, France., Lemaitre J; Center for Immunology of Viral, Auto-immune, Hematological and Bacterial diseases (IMVA-HB/IDMIT), Université Paris-Saclay, Inserm, CEA, Fontenay-aux-Roses, France., Qi W; Novartis Gene Therapies, San Diego, CA, USA., Hudspeth E; Novartis Gene Therapies, NC, USA., Cucalon A; Grousbeck Gene Therapy Center, Schepens Eye Research Institute, Mass Eye and Ear, Boston, MA, USA; Ocular Genomics Institute, Department of Ophthalmology, Harvard Medical School, Boston, MA, USA; The Broad Institute of Harvard and MIT, Cambridge, MA, USA; Harvard Stem Cell Institute, Harvard University, Cambridge, MA, USA., Dyer CD; Gene Therapy Program, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Pampena MB; Gene Therapy Program, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Knox JJ; Department of Pathology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., LaRocque RC; Division of Infectious Diseases, Massachusetts General Hospital, Boston, MA, USA; Department of Medicine, Harvard Medical School, Boston, MA, USA., Charles RC; Division of Infectious Diseases, Massachusetts General Hospital, Boston, MA, USA; Department of Medicine, Harvard Medical School, Boston, MA, USA., Li D; Grousbeck Gene Therapy Center, Schepens Eye Research Institute, Mass Eye and Ear, Boston, MA, USA; Ocular Genomics Institute, Department of Ophthalmology, Harvard Medical School, Boston, MA, USA; The Broad Institute of Harvard and MIT, Cambridge, MA, USA; Harvard Stem Cell Institute, Harvard University, Cambridge, MA, USA., Kim M; Grousbeck Gene Therapy Center, Schepens Eye Research Institute, Mass Eye and Ear, Boston, MA, USA; Ocular Genomics Institute, Department of Ophthalmology, Harvard Medical School, Boston, MA, USA; The Broad Institute of Harvard and MIT, Cambridge, MA, USA; Harvard Stem Cell Institute, Harvard University, Cambridge, MA, USA., Sheridan A; Grousbeck Gene Therapy Center, Schepens Eye Research Institute, Mass Eye and Ear, Boston, MA, USA; Ocular Genomics Institute, Department of Ophthalmology, Harvard Medical School, Boston, MA, USA; The Broad Institute of Harvard and MIT, Cambridge, MA, USA; Harvard Stem Cell Institute, Harvard University, Cambridge, MA, USA., Storm N; Department of Microbiology and National Emerging Infectious Diseases Laboratories, Boston University School of Medicine, Boston, MA 02118, USA., Johnson RI; Department of Microbiology and National Emerging Infectious Diseases Laboratories, Boston University School of Medicine, Boston, MA 02118, USA., Feldman J; Ragon Institute of MGH, MIT, and Harvard, Cambridge, MA 02139, USA., Hauser BM; Ragon Institute of MGH, MIT, and Harvard, Cambridge, MA 02139, USA., Contreras V; Center for Immunology of Viral, Auto-immune, Hematological and Bacterial diseases (IMVA-HB/IDMIT), Université Paris-Saclay, Inserm, CEA, Fontenay-aux-Roses, France., Marlin R; Center for Immunology of Viral, Auto-immune, Hematological and Bacterial diseases (IMVA-HB/IDMIT), Université Paris-Saclay, Inserm, CEA, Fontenay-aux-Roses, France., Tsong Fang RH; Center for Immunology of Viral, Auto-immune, Hematological and Bacterial diseases (IMVA-HB/IDMIT), Université Paris-Saclay, Inserm, CEA, Fontenay-aux-Roses, France., Chapon C; Center for Immunology of Viral, Auto-immune, Hematological and Bacterial diseases (IMVA-HB/IDMIT), Université Paris-Saclay, Inserm, CEA, Fontenay-aux-Roses, France., van der Werf S; Molecular Genetics of RNA Viruses, Department of Virology, Institut Pasteur, CNRS UMR 3569, Université de Paris, Paris, France; National Reference Center for Respiratory Viruses, Institut Pasteur, Paris, France., Zinn E; Grousbeck Gene Therapy Center, Schepens Eye Research Institute, Mass Eye and Ear, Boston, MA, USA; Ocular Genomics Institute, Department of Ophthalmology, Harvard Medical School, Boston, MA, USA; The Broad Institute of Harvard and MIT, Cambridge, MA, USA; Harvard Stem Cell Institute, Harvard University, Cambridge, MA, USA., Ryan A; Grousbeck Gene Therapy Center, Schepens Eye Research Institute, Mass Eye and Ear, Boston, MA, USA; Ocular Genomics Institute, Department of Ophthalmology, Harvard Medical School, Boston, MA, USA; The Broad Institute of Harvard and MIT, Cambridge, MA, USA; Harvard Stem Cell Institute, Harvard University, Cambridge, MA, USA., Kobayashi DT; Translational Innovation Fund, Mass General Brigham Innovation, Cambridge, MA, USA., Chauhan R; Grousbeck Gene Therapy Center, Schepens Eye Research Institute, Mass Eye and Ear, Boston, MA, USA; Ocular Genomics Institute, Department of Ophthalmology, Harvard Medical School, Boston, MA, USA; The Broad Institute of Harvard and MIT, Cambridge, MA, USA; Harvard Stem Cell Institute, Harvard University, Cambridge, MA, USA., McGlynn M; Gene Therapy Program, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Ryan ET; Division of Infectious Diseases, Massachusetts General Hospital, Boston, MA, USA; Department of Medicine, Harvard Medical School, Boston, MA, USA; Department of Immunology and Infectious Diseases, Harvard T. H. Chan School of Public Health, Boston, MA, USA., Schmidt AG; Ragon Institute of MGH, MIT, and Harvard, Cambridge, MA 02139, USA; Department of Microbiology, Harvard Medical School, Boston, MA, USA., Price B; Albamunity, Boston, MA, USA., Honko A; Department of Microbiology and National Emerging Infectious Diseases Laboratories, Boston University School of Medicine, Boston, MA 02118, USA., Griffiths A; Department of Microbiology and National Emerging Infectious Diseases Laboratories, Boston University School of Medicine, Boston, MA 02118, USA., Yaghmour S; Novartis Gene Therapies, San Diego, CA, USA., Hodge R; Novartis Gene Therapies, Libertyville, IL, USA., Betts MR; Gene Therapy Program, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Freeman MW; Center for Computational & Integrative Biology, Department of Medicine, and Translational Research Center, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA., Wilson JM; Gene Therapy Program, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Le Grand R; Center for Immunology of Viral, Auto-immune, Hematological and Bacterial diseases (IMVA-HB/IDMIT), Université Paris-Saclay, Inserm, CEA, Fontenay-aux-Roses, France. Electronic address: roger.le-grand@cea.fr., Vandenberghe LH; Grousbeck Gene Therapy Center, Schepens Eye Research Institute, Mass Eye and Ear, Boston, MA, USA; Ocular Genomics Institute, Department of Ophthalmology, Harvard Medical School, Boston, MA, USA; The Broad Institute of Harvard and MIT, Cambridge, MA, USA; Harvard Stem Cell Institute, Harvard University, Cambridge, MA, USA. Electronic address: luk_vandenberghe@meei.harvard.edu.
Publikováno v:
Cell host & microbe [Cell Host Microbe] 2021 Sep 08; Vol. 29 (9), pp. 1437-1453.e8. Date of Electronic Publication: 2021 Aug 07.
Autor:
Zabaleta N, Dai W, Bhatt U, Chichester JA, Sanmiguel J, Estelien R, Michalson KT, Diop C, Maciorowski D, Qi W, Hudspeth E, Cucalon A, Dyer CD, Pampena MB, Knox JJ, LaRocque RC, Charles RC, Li D, Kim M, Sheridan A, Storm N, Johnson RI, Feldman J, Hauser BM, Zinn E, Ryan A, Kobayashi DT, Chauhan R, McGlynn M, Ryan ET, Schmidt AG, Price B, Honko A, Griffiths A, Yaghmour S, Hodge R, Betts MR, Freeman MW, Wilson JM, Vandenberghe LH
Publikováno v:
BioRxiv : the preprint server for biology [bioRxiv] 2021 Jan 19. Date of Electronic Publication: 2021 Jan 19.
Autor:
Arnold WD; Department of Neurology, The Ohio State University Wexner Medical Center, Columbus Ohio, United States of America.; Department of Physical Medicine and Rehabilitation, The Ohio State University Wexner Medical Center, Columbus Ohio, United States of America., Duque S; VIB Center for the Biology of Disease - KU Leuven Department of Human Genetics, Leuven Belgium, United States of America., Iyer CC; Department of Biological Chemistry and Pharmacology, The Ohio State University Wexner Medical Center, Columbus Ohio, United States of America., Zaworski P; PharmOptima, Portage, Michigan, United States of America., McGovern VL; Department of Biological Chemistry and Pharmacology, The Ohio State University Wexner Medical Center, Columbus Ohio, United States of America., Taylor SJ; PharmOptima, Portage, Michigan, United States of America., von Herrmann KM; SMA Foundation, New York, New York, United States of America., Kobayashi DT; SMA Foundation, New York, New York, United States of America., Chen KS; SMA Foundation, New York, New York, United States of America., Kolb SJ; Department of Neurology, The Ohio State University Wexner Medical Center, Columbus Ohio, United States of America.; Department of Biological Chemistry and Pharmacology, The Ohio State University Wexner Medical Center, Columbus Ohio, United States of America., Paushkin SV; SMA Foundation, New York, New York, United States of America., Burghes AH; Department of Neurology, The Ohio State University Wexner Medical Center, Columbus Ohio, United States of America.; Department of Biological Chemistry and Pharmacology, The Ohio State University Wexner Medical Center, Columbus Ohio, United States of America.
Publikováno v:
PloS one [PLoS One] 2016 Dec 01; Vol. 11 (12), pp. e0167077. Date of Electronic Publication: 2016 Dec 01 (Print Publication: 2016).
Autor:
Cherry JJ; 1 Pfizer, Inc. , Cambridge, Massachusetts., Kobayashi DT, Lynes MM, Naryshkin NN, Tiziano FD, Zaworski PG, Rubin LL, Jarecki J
Publikováno v:
Assay and drug development technologies [Assay Drug Dev Technol] 2014 Aug; Vol. 12 (6), pp. 315-41.
Autor:
Kobayashi DT; Spinal Muscular Atrophy Foundation, New York, New York, United States of America. dkobayashi@smafoundation.org, Shi J, Stephen L, Ballard KL, Dewey R, Mapes J, Chung B, McCarthy K, Swoboda KJ, Crawford TO, Li R, Plasterer T, Joyce C, Chung WK, Kaufmann P, Darras BT, Finkel RS, Sproule DM, Martens WB, McDermott MP, De Vivo DC, Walker MG, Chen KS
Publikováno v:
PloS one [PLoS One] 2013; Vol. 8 (4), pp. e60113. Date of Electronic Publication: 2013 Apr 02.
Autor:
Crawford TO; Departments of Neurology and Pediatrics, The Johns Hopkins University, Baltimore, Maryland, United States of America. tcrawfo@jhmi.edu, Paushkin SV, Kobayashi DT, Forrest SJ, Joyce CL, Finkel RS, Kaufmann P, Swoboda KJ, Tiziano D, Lomastro R, Li RH, Trachtenberg FL, Plasterer T, Chen KS
Publikováno v:
PloS one [PLoS One] 2012; Vol. 7 (4), pp. e33572. Date of Electronic Publication: 2012 Apr 27.
Autor:
Kobayashi DT; Spinal Muscular Atrophy Foundation, New York, New York, United States of America. dkobayashi@smafoundation.org, Decker D, Zaworski P, Klott K, McGonigal J, Ghazal N, Sly L, Chung B, Vanderlugt J, Chen KS
Publikováno v:
PloS one [PLoS One] 2012; Vol. 7 (11), pp. e50763. Date of Electronic Publication: 2012 Nov 30.
Autor:
Finkel RS; Department of Neurology and Pediatrics, The Children's Hospital of Philadelphia and The University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania, United States of America. finkel@email.chop.edu, Crawford TO, Swoboda KJ, Kaufmann P, Juhasz P, Li X, Guo Y, Li RH, Trachtenberg F, Forrest SJ, Kobayashi DT, Chen KS, Joyce CL, Plasterer T
Publikováno v:
PloS one [PLoS One] 2012; Vol. 7 (4), pp. e35462. Date of Electronic Publication: 2012 Apr 27.
Autor:
Kobayashi DT; Spinal Muscular Atrophy Foundation, New York, New York, United States of America. dkobayashi@smafoundation.org, Olson RJ, Sly L, Swanson CJ, Chung B, Naryshkin N, Narasimhan J, Bhattacharyya A, Mullenix M, Chen KS
Publikováno v:
PloS one [PLoS One] 2011; Vol. 6 (8), pp. e24269. Date of Electronic Publication: 2011 Aug 31.