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Optimization of Retinal Gene Therapy for X-Linked Retinitis Pigmentosa Due to RPGR Mutations.
Beltran, William A; Cideciyan, Artur V; Boye, Shannon E; Ye, Guo-Jie; Iwabe, Simone; Dufour, Valerie L; Marinho, Luis Felipe; Swider, Malgorzata; Kosyk, Mychajlo S; Sha, Jin; Boye, Sanford L; Peterson, James J; Witherspoon, C Douglas; Alexander, John J; Ying, Gui-Shuang; Shearman, Mark S; Chulay, Jeffrey D; Hauswirth, William W; Gamlin, Paul D; Jacobson, Samuel G; Aguirre, Gustavo D.
Afiliación
  • Beltran WA; Division of Experimental Retinal Therapies, Department of Clinical Studies, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19014, USA. Electronic address: wbeltran@vet.upenn.edu.
  • Cideciyan AV; Scheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
  • Boye SE; Department of Ophthalmology, College of Medicine, University of Florida, Gainesville, FL 32610, USA.
  • Ye GJ; Applied Genetic Technologies Corporation, Alachua, FL 32615, USA.
  • Iwabe S; Division of Experimental Retinal Therapies, Department of Clinical Studies, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19014, USA.
  • Dufour VL; Division of Experimental Retinal Therapies, Department of Clinical Studies, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19014, USA.
  • Marinho LF; Division of Experimental Retinal Therapies, Department of Clinical Studies, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19014, USA.
  • Swider M; Scheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
  • Kosyk MS; Scheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
  • Sha J; Scheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
  • Boye SL; Department of Ophthalmology, College of Medicine, University of Florida, Gainesville, FL 32610, USA.
  • Peterson JJ; Department of Ophthalmology, College of Medicine, University of Florida, Gainesville, FL 32610, USA.
  • Witherspoon CD; Department of Ophthalmology, School of Medicine, University of Alabama at Birmingham, Birmingham, AL 35294, USA.
  • Alexander JJ; Department of Human Genetics, School of Medicine, Emory University, Atlanta, GA 30303, USA.
  • Ying GS; Scheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
  • Shearman MS; Applied Genetic Technologies Corporation, Alachua, FL 32615, USA.
  • Chulay JD; Applied Genetic Technologies Corporation, Alachua, FL 32615, USA.
  • Hauswirth WW; Department of Ophthalmology, College of Medicine, University of Florida, Gainesville, FL 32610, USA.
  • Gamlin PD; Department of Ophthalmology, School of Medicine, University of Alabama at Birmingham, Birmingham, AL 35294, USA.
  • Jacobson SG; Scheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
  • Aguirre GD; Division of Experimental Retinal Therapies, Department of Clinical Studies, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19014, USA.
Mol Ther ; 25(8): 1866-1880, 2017 08 02.
Article en En | MEDLINE | ID: mdl-28566226
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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Retina / Terapia Genética / Proteínas Portadoras / Retinitis Pigmentosa / Genes Ligados a X / Proteínas del Ojo / Mutación Tipo de estudio: Clinical_trials / Diagnostic_studies / Prognostic_studies Límite: Animals / Humans Idioma: En Revista: Mol Ther Asunto de la revista: BIOLOGIA MOLECULAR / TERAPEUTICA Año: 2017 Tipo del documento: Article
Texto completo
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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Retina / Terapia Genética / Proteínas Portadoras / Retinitis Pigmentosa / Genes Ligados a X / Proteínas del Ojo / Mutación Tipo de estudio: Clinical_trials / Diagnostic_studies / Prognostic_studies Límite: Animals / Humans Idioma: En Revista: Mol Ther Asunto de la revista: BIOLOGIA MOLECULAR / TERAPEUTICA Año: 2017 Tipo del documento: Article