Zobrazeno 1 - 10
of 702
pro vyhledávání: '"Schnell FJ"'
Systemic PPMO-mediated dystrophin expression in the Dup2 mouse model of Duchenne muscular dystrophy.
Autor:
Gushchina LV; The Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA., Vetter TA; The Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA., Frair EC; The Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA., Bradley AJ; The Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA., Grounds KM; The Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA., Lay JW; The Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA., Huang N; The Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA., Suhaiba A; The Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA., Schnell FJ; Sarepta Therapeutics, Inc., Cambridge, MA, USA., Hanson G; Sarepta Therapeutics, Inc., Cambridge, MA, USA., Simmons TR; The Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA., Wein N; The Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA.; Department of Pediatrics, The Ohio State University, Columbus, OH, USA., Flanigan KM; The Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA.; Department of Pediatrics, The Ohio State University, Columbus, OH, USA.; Department of Neurology, The Ohio State University, Columbus, OH, USA.
Publikováno v:
Molecular therapy. Nucleic acids [Mol Ther Nucleic Acids] 2022 Nov 09; Vol. 30, pp. 479-492. Date of Electronic Publication: 2022 Nov 09 (Print Publication: 2022).
Autor:
Shaocong Li1, Tao Li1, Zijie Jiang1, Wenyu Hou1, Qirui Hou1, Serrano, Boris Ramos2, Barcenas, Adileidys Ruiz2, Yuhua Wang1 wangyhsri@126.com, Weiguo Zhao1 wgzsri@126.com
Publikováno v:
Animal Bioscience. Dec2024, Vol. 37 Issue 12, p2101-2112. 13p.
Autor:
Rossi, Rachele1,2 (AUTHOR), Torelli, Silvia1,2 (AUTHOR), Moore, Marc1,2,3 (AUTHOR), Ala, Pierpaolo1,2 (AUTHOR), Morgan, Jennifer1,2 (AUTHOR), Malhotra, Jyoti4 (AUTHOR), Muntoni, Francesco1,2 (AUTHOR) f.muntoni@ucl.ac.uk
Publikováno v:
Skeletal Muscle. 11/29/2024, Vol. 14 Issue 1, p1-14. 14p.
Autor:
Frank DE; From Sarepta Therapeutics (D.E.F., F.J.S., C.A., S.H.E.-H., C.A.D., J.S.C.), Cambridge, MA; University College London (J.M., V.S., J.D., F.M.); Centre of Gene and Cell Therapy and Centre for Biomedical Sciences (G.D.), Royal Holloway, University of London, Egham, Surrey; Newcastle University John Walton Muscular Dystrophy Research Centre and the Newcastle Hospitals NHS Foundation Trust (V.S., M.G.), Newcastle upon Tyne, UK; Paediatric Neurology and Centro Clinico Nemo (E.M.), Catholic University and Policlinico Gemelli, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy; Institute I-Motion (L.S.), Hôpital Armand-Trousseau, Paris, France; Neuromuscular Reference Center (L.S.), CHU Liège, Belgium; Great Ormond Street Hospital (F.M.); and NIHR Great Ormond Street Hospital Biomedical Research Centre (F.M.), London, UK., Schnell FJ; From Sarepta Therapeutics (D.E.F., F.J.S., C.A., S.H.E.-H., C.A.D., J.S.C.), Cambridge, MA; University College London (J.M., V.S., J.D., F.M.); Centre of Gene and Cell Therapy and Centre for Biomedical Sciences (G.D.), Royal Holloway, University of London, Egham, Surrey; Newcastle University John Walton Muscular Dystrophy Research Centre and the Newcastle Hospitals NHS Foundation Trust (V.S., M.G.), Newcastle upon Tyne, UK; Paediatric Neurology and Centro Clinico Nemo (E.M.), Catholic University and Policlinico Gemelli, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy; Institute I-Motion (L.S.), Hôpital Armand-Trousseau, Paris, France; Neuromuscular Reference Center (L.S.), CHU Liège, Belgium; Great Ormond Street Hospital (F.M.); and NIHR Great Ormond Street Hospital Biomedical Research Centre (F.M.), London, UK., Akana C; From Sarepta Therapeutics (D.E.F., F.J.S., C.A., S.H.E.-H., C.A.D., J.S.C.), Cambridge, MA; University College London (J.M., V.S., J.D., F.M.); Centre of Gene and Cell Therapy and Centre for Biomedical Sciences (G.D.), Royal Holloway, University of London, Egham, Surrey; Newcastle University John Walton Muscular Dystrophy Research Centre and the Newcastle Hospitals NHS Foundation Trust (V.S., M.G.), Newcastle upon Tyne, UK; Paediatric Neurology and Centro Clinico Nemo (E.M.), Catholic University and Policlinico Gemelli, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy; Institute I-Motion (L.S.), Hôpital Armand-Trousseau, Paris, France; Neuromuscular Reference Center (L.S.), CHU Liège, Belgium; Great Ormond Street Hospital (F.M.); and NIHR Great Ormond Street Hospital Biomedical Research Centre (F.M.), London, UK., El-Husayni SH; From Sarepta Therapeutics (D.E.F., F.J.S., C.A., S.H.E.-H., C.A.D., J.S.C.), Cambridge, MA; University College London (J.M., V.S., J.D., F.M.); Centre of Gene and Cell Therapy and Centre for Biomedical Sciences (G.D.), Royal Holloway, University of London, Egham, Surrey; Newcastle University John Walton Muscular Dystrophy Research Centre and the Newcastle Hospitals NHS Foundation Trust (V.S., M.G.), Newcastle upon Tyne, UK; Paediatric Neurology and Centro Clinico Nemo (E.M.), Catholic University and Policlinico Gemelli, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy; Institute I-Motion (L.S.), Hôpital Armand-Trousseau, Paris, France; Neuromuscular Reference Center (L.S.), CHU Liège, Belgium; Great Ormond Street Hospital (F.M.); and NIHR Great Ormond Street Hospital Biomedical Research Centre (F.M.), London, UK., Desjardins CA; From Sarepta Therapeutics (D.E.F., F.J.S., C.A., S.H.E.-H., C.A.D., J.S.C.), Cambridge, MA; University College London (J.M., V.S., J.D., F.M.); Centre of Gene and Cell Therapy and Centre for Biomedical Sciences (G.D.), Royal Holloway, University of London, Egham, Surrey; Newcastle University John Walton Muscular Dystrophy Research Centre and the Newcastle Hospitals NHS Foundation Trust (V.S., M.G.), Newcastle upon Tyne, UK; Paediatric Neurology and Centro Clinico Nemo (E.M.), Catholic University and Policlinico Gemelli, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy; Institute I-Motion (L.S.), Hôpital Armand-Trousseau, Paris, France; Neuromuscular Reference Center (L.S.), CHU Liège, Belgium; Great Ormond Street Hospital (F.M.); and NIHR Great Ormond Street Hospital Biomedical Research Centre (F.M.), London, UK., Morgan J; From Sarepta Therapeutics (D.E.F., F.J.S., C.A., S.H.E.-H., C.A.D., J.S.C.), Cambridge, MA; University College London (J.M., V.S., J.D., F.M.); Centre of Gene and Cell Therapy and Centre for Biomedical Sciences (G.D.), Royal Holloway, University of London, Egham, Surrey; Newcastle University John Walton Muscular Dystrophy Research Centre and the Newcastle Hospitals NHS Foundation Trust (V.S., M.G.), Newcastle upon Tyne, UK; Paediatric Neurology and Centro Clinico Nemo (E.M.), Catholic University and Policlinico Gemelli, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy; Institute I-Motion (L.S.), Hôpital Armand-Trousseau, Paris, France; Neuromuscular Reference Center (L.S.), CHU Liège, Belgium; Great Ormond Street Hospital (F.M.); and NIHR Great Ormond Street Hospital Biomedical Research Centre (F.M.), London, UK., Charleston JS; From Sarepta Therapeutics (D.E.F., F.J.S., C.A., S.H.E.-H., C.A.D., J.S.C.), Cambridge, MA; University College London (J.M., V.S., J.D., F.M.); Centre of Gene and Cell Therapy and Centre for Biomedical Sciences (G.D.), Royal Holloway, University of London, Egham, Surrey; Newcastle University John Walton Muscular Dystrophy Research Centre and the Newcastle Hospitals NHS Foundation Trust (V.S., M.G.), Newcastle upon Tyne, UK; Paediatric Neurology and Centro Clinico Nemo (E.M.), Catholic University and Policlinico Gemelli, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy; Institute I-Motion (L.S.), Hôpital Armand-Trousseau, Paris, France; Neuromuscular Reference Center (L.S.), CHU Liège, Belgium; Great Ormond Street Hospital (F.M.); and NIHR Great Ormond Street Hospital Biomedical Research Centre (F.M.), London, UK., Sardone V; From Sarepta Therapeutics (D.E.F., F.J.S., C.A., S.H.E.-H., C.A.D., J.S.C.), Cambridge, MA; University College London (J.M., V.S., J.D., F.M.); Centre of Gene and Cell Therapy and Centre for Biomedical Sciences (G.D.), Royal Holloway, University of London, Egham, Surrey; Newcastle University John Walton Muscular Dystrophy Research Centre and the Newcastle Hospitals NHS Foundation Trust (V.S., M.G.), Newcastle upon Tyne, UK; Paediatric Neurology and Centro Clinico Nemo (E.M.), Catholic University and Policlinico Gemelli, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy; Institute I-Motion (L.S.), Hôpital Armand-Trousseau, Paris, France; Neuromuscular Reference Center (L.S.), CHU Liège, Belgium; Great Ormond Street Hospital (F.M.); and NIHR Great Ormond Street Hospital Biomedical Research Centre (F.M.), London, UK., Domingos J; From Sarepta Therapeutics (D.E.F., F.J.S., C.A., S.H.E.-H., C.A.D., J.S.C.), Cambridge, MA; University College London (J.M., V.S., J.D., F.M.); Centre of Gene and Cell Therapy and Centre for Biomedical Sciences (G.D.), Royal Holloway, University of London, Egham, Surrey; Newcastle University John Walton Muscular Dystrophy Research Centre and the Newcastle Hospitals NHS Foundation Trust (V.S., M.G.), Newcastle upon Tyne, UK; Paediatric Neurology and Centro Clinico Nemo (E.M.), Catholic University and Policlinico Gemelli, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy; Institute I-Motion (L.S.), Hôpital Armand-Trousseau, Paris, France; Neuromuscular Reference Center (L.S.), CHU Liège, Belgium; Great Ormond Street Hospital (F.M.); and NIHR Great Ormond Street Hospital Biomedical Research Centre (F.M.), London, UK., Dickson G; From Sarepta Therapeutics (D.E.F., F.J.S., C.A., S.H.E.-H., C.A.D., J.S.C.), Cambridge, MA; University College London (J.M., V.S., J.D., F.M.); Centre of Gene and Cell Therapy and Centre for Biomedical Sciences (G.D.), Royal Holloway, University of London, Egham, Surrey; Newcastle University John Walton Muscular Dystrophy Research Centre and the Newcastle Hospitals NHS Foundation Trust (V.S., M.G.), Newcastle upon Tyne, UK; Paediatric Neurology and Centro Clinico Nemo (E.M.), Catholic University and Policlinico Gemelli, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy; Institute I-Motion (L.S.), Hôpital Armand-Trousseau, Paris, France; Neuromuscular Reference Center (L.S.), CHU Liège, Belgium; Great Ormond Street Hospital (F.M.); and NIHR Great Ormond Street Hospital Biomedical Research Centre (F.M.), London, UK., Straub V; From Sarepta Therapeutics (D.E.F., F.J.S., C.A., S.H.E.-H., C.A.D., J.S.C.), Cambridge, MA; University College London (J.M., V.S., J.D., F.M.); Centre of Gene and Cell Therapy and Centre for Biomedical Sciences (G.D.), Royal Holloway, University of London, Egham, Surrey; Newcastle University John Walton Muscular Dystrophy Research Centre and the Newcastle Hospitals NHS Foundation Trust (V.S., M.G.), Newcastle upon Tyne, UK; Paediatric Neurology and Centro Clinico Nemo (E.M.), Catholic University and Policlinico Gemelli, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy; Institute I-Motion (L.S.), Hôpital Armand-Trousseau, Paris, France; Neuromuscular Reference Center (L.S.), CHU Liège, Belgium; Great Ormond Street Hospital (F.M.); and NIHR Great Ormond Street Hospital Biomedical Research Centre (F.M.), London, UK., Guglieri M; From Sarepta Therapeutics (D.E.F., F.J.S., C.A., S.H.E.-H., C.A.D., J.S.C.), Cambridge, MA; University College London (J.M., V.S., J.D., F.M.); Centre of Gene and Cell Therapy and Centre for Biomedical Sciences (G.D.), Royal Holloway, University of London, Egham, Surrey; Newcastle University John Walton Muscular Dystrophy Research Centre and the Newcastle Hospitals NHS Foundation Trust (V.S., M.G.), Newcastle upon Tyne, UK; Paediatric Neurology and Centro Clinico Nemo (E.M.), Catholic University and Policlinico Gemelli, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy; Institute I-Motion (L.S.), Hôpital Armand-Trousseau, Paris, France; Neuromuscular Reference Center (L.S.), CHU Liège, Belgium; Great Ormond Street Hospital (F.M.); and NIHR Great Ormond Street Hospital Biomedical Research Centre (F.M.), London, UK., Mercuri E; From Sarepta Therapeutics (D.E.F., F.J.S., C.A., S.H.E.-H., C.A.D., J.S.C.), Cambridge, MA; University College London (J.M., V.S., J.D., F.M.); Centre of Gene and Cell Therapy and Centre for Biomedical Sciences (G.D.), Royal Holloway, University of London, Egham, Surrey; Newcastle University John Walton Muscular Dystrophy Research Centre and the Newcastle Hospitals NHS Foundation Trust (V.S., M.G.), Newcastle upon Tyne, UK; Paediatric Neurology and Centro Clinico Nemo (E.M.), Catholic University and Policlinico Gemelli, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy; Institute I-Motion (L.S.), Hôpital Armand-Trousseau, Paris, France; Neuromuscular Reference Center (L.S.), CHU Liège, Belgium; Great Ormond Street Hospital (F.M.); and NIHR Great Ormond Street Hospital Biomedical Research Centre (F.M.), London, UK., Servais L; From Sarepta Therapeutics (D.E.F., F.J.S., C.A., S.H.E.-H., C.A.D., J.S.C.), Cambridge, MA; University College London (J.M., V.S., J.D., F.M.); Centre of Gene and Cell Therapy and Centre for Biomedical Sciences (G.D.), Royal Holloway, University of London, Egham, Surrey; Newcastle University John Walton Muscular Dystrophy Research Centre and the Newcastle Hospitals NHS Foundation Trust (V.S., M.G.), Newcastle upon Tyne, UK; Paediatric Neurology and Centro Clinico Nemo (E.M.), Catholic University and Policlinico Gemelli, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy; Institute I-Motion (L.S.), Hôpital Armand-Trousseau, Paris, France; Neuromuscular Reference Center (L.S.), CHU Liège, Belgium; Great Ormond Street Hospital (F.M.); and NIHR Great Ormond Street Hospital Biomedical Research Centre (F.M.), London, UK., Muntoni F; From Sarepta Therapeutics (D.E.F., F.J.S., C.A., S.H.E.-H., C.A.D., J.S.C.), Cambridge, MA; University College London (J.M., V.S., J.D., F.M.); Centre of Gene and Cell Therapy and Centre for Biomedical Sciences (G.D.), Royal Holloway, University of London, Egham, Surrey; Newcastle University John Walton Muscular Dystrophy Research Centre and the Newcastle Hospitals NHS Foundation Trust (V.S., M.G.), Newcastle upon Tyne, UK; Paediatric Neurology and Centro Clinico Nemo (E.M.), Catholic University and Policlinico Gemelli, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy; Institute I-Motion (L.S.), Hôpital Armand-Trousseau, Paris, France; Neuromuscular Reference Center (L.S.), CHU Liège, Belgium; Great Ormond Street Hospital (F.M.); and NIHR Great Ormond Street Hospital Biomedical Research Centre (F.M.), London, UK. f.muntoni@ucl.ac.uk.
Publikováno v:
Neurology [Neurology] 2020 May 26; Vol. 94 (21), pp. e2270-e2282. Date of Electronic Publication: 2020 Mar 05.
Autor:
Aung-Htut MT; Centre for Molecular Medicine and Innovative Therapeutics, Murdoch University, Murdoch, 6150, Australia.; Perron Institute for Neurological and Translational Science and Centre for Neuromuscular and Neurological Disorders, The University of Western Australia, Perth, 6009, Australia., Ham KA; Centre for Molecular Medicine and Innovative Therapeutics, Murdoch University, Murdoch, 6150, Australia.; Perron Institute for Neurological and Translational Science and Centre for Neuromuscular and Neurological Disorders, The University of Western Australia, Perth, 6009, Australia., Tchan M; Genetic Medicine, Westmead Hospital, Sydney, 2145, Australia.; Sydney Medical School, The University of Sydney, Sydney, 2006, Australia., Johnsen R; Centre for Molecular Medicine and Innovative Therapeutics, Murdoch University, Murdoch, 6150, Australia., Schnell FJ; Sarepta Therapeutics, Cambridge, 02142, USA., Fletcher S; Centre for Molecular Medicine and Innovative Therapeutics, Murdoch University, Murdoch, 6150, Australia. s.fletcher@murdoch.edu.au.; Perron Institute for Neurological and Translational Science and Centre for Neuromuscular and Neurological Disorders, The University of Western Australia, Perth, 6009, Australia. s.fletcher@murdoch.edu.au., Wilton SD; Centre for Molecular Medicine and Innovative Therapeutics, Murdoch University, Murdoch, 6150, Australia. s.wilton@murdoch.edu.au.; Perron Institute for Neurological and Translational Science and Centre for Neuromuscular and Neurological Disorders, The University of Western Australia, Perth, 6009, Australia. s.wilton@murdoch.edu.au.
Publikováno v:
Scientific reports [Sci Rep] 2020 Apr 21; Vol. 10 (1), pp. 6702. Date of Electronic Publication: 2020 Apr 21.
Autor:
Bourgeois Yoshioka, Clémence Kiho1,2 (AUTHOR), Takenaka-Ninagawa, Nana1,3 (AUTHOR) nana.takenaka@cira.kyoto-u.ac.jp, Goto, Megumi1 (AUTHOR), Miki, Mayuho1,2 (AUTHOR), Watanabe, Daiki4,5 (AUTHOR), Yamamoto, Masamichi5 (AUTHOR), Aoyama, Tomoki2 (AUTHOR), Sakurai, Hidetoshi1 (AUTHOR) hsakurai@cira.kyoto-u.ac.jp
Publikováno v:
Stem Cell Research & Therapy. 9/19/2024, Vol. 15 Issue 1, p1-24. 24p.
Autor:
Lu-Nguyen N; Centres of Gene and Cell Therapy and Biomedical Sciences, School of Biological Sciences, Royal Holloway-University of London, Egham, Surrey, TW20 0EX, UK., Ferry A; Sorbonne Université UMRS974 INSERM, Institut de Myologie, 75013 Paris, France.; Université Sorbonne Paris Cité, 75006 Paris, France., Schnell FJ; Sarepta Therapeutics, Inc., 215 First Street, Cambridge, MA 02142, USA., Hanson GJ; Sarepta Therapeutics, Inc., 215 First Street, Cambridge, MA 02142, USA., Popplewell L; Centres of Gene and Cell Therapy and Biomedical Sciences, School of Biological Sciences, Royal Holloway-University of London, Egham, Surrey, TW20 0EX, UK., Dickson G; Centres of Gene and Cell Therapy and Biomedical Sciences, School of Biological Sciences, Royal Holloway-University of London, Egham, Surrey, TW20 0EX, UK., Malerba A; Centres of Gene and Cell Therapy and Biomedical Sciences, School of Biological Sciences, Royal Holloway-University of London, Egham, Surrey, TW20 0EX, UK.
Publikováno v:
Human molecular genetics [Hum Mol Genet] 2019 Sep 15; Vol. 28 (18), pp. 3091-3100.
Autor:
Alfano LN; Pediatrics, Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH., Charleston JS; Sarepta Therapeutics, Inc., Cambridge, MA., Connolly AM; Currently: Pediatrics, Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH.; Department of Neurology, Washington University School of Medicine, St. Louis, MO., Cripe L; Pediatrics, Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH., Donoghue C; Sarepta Therapeutics, Inc., Cambridge, MA., Dracker R; Summerwood Pediatrics/Infusacare Medical Services, PC, Liverpool, NY., Dworzak J; Sarepta Therapeutics, Inc., Cambridge, MA., Eliopoulos H; Sarepta Therapeutics, Inc., Cambridge, MA., Frank DE; Sarepta Therapeutics, Inc., Cambridge, MA., Lewis S; Pediatrics, Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH., Lucas K; Sarepta Therapeutics, Inc., Cambridge, MA., Lynch J; Sarepta Therapeutics, Inc., Cambridge, MA., Milici AJ; Flagship Biosciences, Westminster, CO., Flynt A; PharPoint Research, Durham, NC., Naughton E; Sarepta Therapeutics, Inc., Cambridge, MA., Rodino-Klapac LR; Pediatrics, Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH.; Currently: Sarepta Therapeutics, Inc., Cambridge, MA, USA., Sahenk Z; Pediatrics, Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH., Schnell FJ; Sarepta Therapeutics, Inc., Cambridge, MA., Young GD; Flagship Biosciences, Westminster, CO., Mendell JR; Pediatrics, Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH., Lowes LP; Pediatrics, Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH.
Publikováno v:
Medicine [Medicine (Baltimore)] 2019 Jun; Vol. 98 (26), pp. e15858.
Autor:
Charleston JS; From Sarepta Therapeutics, Inc (J.S.C., F.J.S., J.D., C.D., J.V., U.D., B.W., D.F.), Cambridge, MA; Nationwide Children's Hospital (S.L., L.C., L.R.R.-K., Z.S., J.R.M.), Columbus, OH; and Flagship Biosciences (G.D.Y., A.J.M.), Westminster, CO. jcharleston@sarepta.com., Schnell FJ; From Sarepta Therapeutics, Inc (J.S.C., F.J.S., J.D., C.D., J.V., U.D., B.W., D.F.), Cambridge, MA; Nationwide Children's Hospital (S.L., L.C., L.R.R.-K., Z.S., J.R.M.), Columbus, OH; and Flagship Biosciences (G.D.Y., A.J.M.), Westminster, CO., Dworzak J; From Sarepta Therapeutics, Inc (J.S.C., F.J.S., J.D., C.D., J.V., U.D., B.W., D.F.), Cambridge, MA; Nationwide Children's Hospital (S.L., L.C., L.R.R.-K., Z.S., J.R.M.), Columbus, OH; and Flagship Biosciences (G.D.Y., A.J.M.), Westminster, CO., Donoghue C; From Sarepta Therapeutics, Inc (J.S.C., F.J.S., J.D., C.D., J.V., U.D., B.W., D.F.), Cambridge, MA; Nationwide Children's Hospital (S.L., L.C., L.R.R.-K., Z.S., J.R.M.), Columbus, OH; and Flagship Biosciences (G.D.Y., A.J.M.), Westminster, CO., Lewis S; From Sarepta Therapeutics, Inc (J.S.C., F.J.S., J.D., C.D., J.V., U.D., B.W., D.F.), Cambridge, MA; Nationwide Children's Hospital (S.L., L.C., L.R.R.-K., Z.S., J.R.M.), Columbus, OH; and Flagship Biosciences (G.D.Y., A.J.M.), Westminster, CO., Chen L; From Sarepta Therapeutics, Inc (J.S.C., F.J.S., J.D., C.D., J.V., U.D., B.W., D.F.), Cambridge, MA; Nationwide Children's Hospital (S.L., L.C., L.R.R.-K., Z.S., J.R.M.), Columbus, OH; and Flagship Biosciences (G.D.Y., A.J.M.), Westminster, CO., Young GD; From Sarepta Therapeutics, Inc (J.S.C., F.J.S., J.D., C.D., J.V., U.D., B.W., D.F.), Cambridge, MA; Nationwide Children's Hospital (S.L., L.C., L.R.R.-K., Z.S., J.R.M.), Columbus, OH; and Flagship Biosciences (G.D.Y., A.J.M.), Westminster, CO., Milici AJ; From Sarepta Therapeutics, Inc (J.S.C., F.J.S., J.D., C.D., J.V., U.D., B.W., D.F.), Cambridge, MA; Nationwide Children's Hospital (S.L., L.C., L.R.R.-K., Z.S., J.R.M.), Columbus, OH; and Flagship Biosciences (G.D.Y., A.J.M.), Westminster, CO., Voss J; From Sarepta Therapeutics, Inc (J.S.C., F.J.S., J.D., C.D., J.V., U.D., B.W., D.F.), Cambridge, MA; Nationwide Children's Hospital (S.L., L.C., L.R.R.-K., Z.S., J.R.M.), Columbus, OH; and Flagship Biosciences (G.D.Y., A.J.M.), Westminster, CO., DeAlwis U; From Sarepta Therapeutics, Inc (J.S.C., F.J.S., J.D., C.D., J.V., U.D., B.W., D.F.), Cambridge, MA; Nationwide Children's Hospital (S.L., L.C., L.R.R.-K., Z.S., J.R.M.), Columbus, OH; and Flagship Biosciences (G.D.Y., A.J.M.), Westminster, CO., Wentworth B; From Sarepta Therapeutics, Inc (J.S.C., F.J.S., J.D., C.D., J.V., U.D., B.W., D.F.), Cambridge, MA; Nationwide Children's Hospital (S.L., L.C., L.R.R.-K., Z.S., J.R.M.), Columbus, OH; and Flagship Biosciences (G.D.Y., A.J.M.), Westminster, CO., Rodino-Klapac LR; From Sarepta Therapeutics, Inc (J.S.C., F.J.S., J.D., C.D., J.V., U.D., B.W., D.F.), Cambridge, MA; Nationwide Children's Hospital (S.L., L.C., L.R.R.-K., Z.S., J.R.M.), Columbus, OH; and Flagship Biosciences (G.D.Y., A.J.M.), Westminster, CO., Sahenk Z; From Sarepta Therapeutics, Inc (J.S.C., F.J.S., J.D., C.D., J.V., U.D., B.W., D.F.), Cambridge, MA; Nationwide Children's Hospital (S.L., L.C., L.R.R.-K., Z.S., J.R.M.), Columbus, OH; and Flagship Biosciences (G.D.Y., A.J.M.), Westminster, CO., Frank D; From Sarepta Therapeutics, Inc (J.S.C., F.J.S., J.D., C.D., J.V., U.D., B.W., D.F.), Cambridge, MA; Nationwide Children's Hospital (S.L., L.C., L.R.R.-K., Z.S., J.R.M.), Columbus, OH; and Flagship Biosciences (G.D.Y., A.J.M.), Westminster, CO., Mendell JR; From Sarepta Therapeutics, Inc (J.S.C., F.J.S., J.D., C.D., J.V., U.D., B.W., D.F.), Cambridge, MA; Nationwide Children's Hospital (S.L., L.C., L.R.R.-K., Z.S., J.R.M.), Columbus, OH; and Flagship Biosciences (G.D.Y., A.J.M.), Westminster, CO.
Publikováno v:
Neurology [Neurology] 2018 Jun 12; Vol. 90 (24), pp. e2146-e2154. Date of Electronic Publication: 2018 May 11.
Autor:
Kelly, Jessica B.1 (AUTHOR), Nolan, Aaron C.1 (AUTHOR), Zeden, Merve S.1 (AUTHOR) merve.zeden@universityofgalway.ie
Publikováno v:
PLoS Pathogens. 6/13/2024, Vol. 20 Issue 6, p1-9. 9p.