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Intrastromal Gene Therapy Prevents and Reverses Advanced Corneal Clouding in a Canine Model of Mucopolysaccharidosis I.
Miyadera, Keiko; Conatser, Laura; Llanga, Telmo A; Carlin, Kendall; O'Donnell, Patricia; Bagel, Jessica; Song, Liujiang; Kurtzberg, Joanne; Samulski, R Jude; Gilger, Brian; Hirsch, Matthew L.
Afiliação
  • Miyadera K; Section of Ophthalmology, Department of Clinical Sciences and Advanced Medicine, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA, USA.
  • Conatser L; Gene Therapy Center, School of Medicine, University of North Carolina, Chapel Hill, NC, USA; Department of Ophthalmology, University of North Carolina, Chapel Hill, NC 27599, USA.
  • Llanga TA; Gene Therapy Center, School of Medicine, University of North Carolina, Chapel Hill, NC, USA; Department of Ophthalmology, University of North Carolina, Chapel Hill, NC 27599, USA.
  • Carlin K; Section of Ophthalmology, Department of Clinical Sciences and Advanced Medicine, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA, USA.
  • O'Donnell P; Section of Neurology and Neurosurgery, Department of Clinical Sciences and Advanced Medicine, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA, USA.
  • Bagel J; Section of Neurology and Neurosurgery, Department of Clinical Sciences and Advanced Medicine, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA, USA.
  • Song L; Gene Therapy Center, School of Medicine, University of North Carolina, Chapel Hill, NC, USA; Department of Ophthalmology, University of North Carolina, Chapel Hill, NC 27599, USA.
  • Kurtzberg J; Department of Pediatrics, Duke University, Durham, NC, USA.
  • Samulski RJ; Gene Therapy Center, School of Medicine, University of North Carolina, Chapel Hill, NC, USA.
  • Gilger B; Department of Clinical Sciences, North Carolina State University, Raleigh, NC 27607, USA.
  • Hirsch ML; Gene Therapy Center, School of Medicine, University of North Carolina, Chapel Hill, NC, USA; Department of Ophthalmology, University of North Carolina, Chapel Hill, NC 27599, USA. Electronic address: mhirsch@email.unc.edu.
Mol Ther ; 28(6): 1455-1463, 2020 06 03.
Article em En | MEDLINE | ID: mdl-32330426
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Terapia Genética / Mucopolissacaridose I / Técnicas de Transferência de Genes / Doenças da Córnea Tipo de estudo: Diagnostic_studies / Prognostic_studies Limite: Animals Idioma: En Revista: Mol Ther Assunto da revista: BIOLOGIA MOLECULAR / TERAPEUTICA Ano de publicação: 2020 Tipo de documento: Article País de afiliação: Estados Unidos País de publicação: Estados Unidos
Texto completo
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XML
PubMed Links
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Terapia Genética / Mucopolissacaridose I / Técnicas de Transferência de Genes / Doenças da Córnea Tipo de estudo: Diagnostic_studies / Prognostic_studies Limite: Animals Idioma: En Revista: Mol Ther Assunto da revista: BIOLOGIA MOLECULAR / TERAPEUTICA Ano de publicação: 2020 Tipo de documento: Article País de afiliação: Estados Unidos País de publicação: Estados Unidos