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Safety and improved efficacy signals following gene therapy in childhood blindness caused by GUCY2D mutations.
Jacobson, Samuel G; Cideciyan, Artur V; Ho, Allen C; Peshenko, Igor V; Garafalo, Alexandra V; Roman, Alejandro J; Sumaroka, Alexander; Wu, Vivian; Krishnan, Arun K; Sheplock, Rebecca; Boye, Sanford L; Dizhoor, Alexander M; Boye, Shannon E.
Afiliação
  • Jacobson SG; Scheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
  • Cideciyan AV; Scheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
  • Ho AC; Wills Eye Hospital, Thomas Jefferson University, Philadelphia, PA, USA.
  • Peshenko IV; Pennsylvania College of Optometry, Salus University, Elkins Park, PA, USA.
  • Garafalo AV; Scheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
  • Roman AJ; Scheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
  • Sumaroka A; Scheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
  • Wu V; Scheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
  • Krishnan AK; Scheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
  • Sheplock R; Scheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
  • Boye SL; Department of Pediatrics, Powell Gene Therapy Center, University of Florida College of Medicine, Gainesville, FL, USA.
  • Dizhoor AM; Pennsylvania College of Optometry, Salus University, Elkins Park, PA, USA.
  • Boye SE; Department of Pediatrics, Division of Cellular and Molecular Therapy, University of Florida College of Medicine, Gainesville, FL, USA.
iScience ; 24(5): 102409, 2021 May 21.
Article em En | MEDLINE | ID: mdl-33997691
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Idioma: En Ano de publicação: 2021 Tipo de documento: Article
Texto completo
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PubMed Links
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Idioma: En Ano de publicação: 2021 Tipo de documento: Article