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Toxicity and Efficacy Evaluation of an Adeno-Associated Virus Vector Expressing Codon-Optimized RPGR Delivered by Subretinal Injection in a Canine Model of X-linked Retinitis Pigmentosa.
Dufour, Valérie L; Cideciyan, Artur V; Ye, Guo-Jie; Song, Chunjuan; Timmers, Adrian; Habecker, Perry L; Pan, Wei; Weinstein, Nicole M; Swider, Malgorzata; Durham, Amy C; Ying, Gui-Shuang; Robinson, Paulette M; Jacobson, Samuel G; Knop, David R; Chulay, Jeffrey D; Shearman, Mark S; Aguirre, Gustavo D; Beltran, William A.
Affiliation
  • Dufour VL; Division of Experimental Retinal Therapies, Department of Clinical Sciences & Advanced Medicine, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.
  • Cideciyan AV; Department of Ophthalmology, Scheie Eye Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.
  • Ye GJ; Applied Genetic Technologies Corporation, Alachua, Florida.
  • Song C; Applied Genetic Technologies Corporation, Alachua, Florida.
  • Timmers A; Applied Genetic Technologies Corporation, Alachua, Florida.
  • Habecker PL; Department of Pathobiology, New Bolton Center, School of Veterinary Medicine, University of Pennsylvania, Kennett Square, Pennsylvania.
  • Pan W; Department of Ophthalmology, Scheie Eye Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.
  • Weinstein NM; Department of Pathobiology, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.
  • Swider M; Department of Ophthalmology, Scheie Eye Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.
  • Durham AC; Department of Pathobiology, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.
  • Ying GS; Department of Ophthalmology, Scheie Eye Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.
  • Robinson PM; Applied Genetic Technologies Corporation, Alachua, Florida.
  • Jacobson SG; Department of Ophthalmology, Scheie Eye Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.
  • Knop DR; Applied Genetic Technologies Corporation, Alachua, Florida.
  • Chulay JD; Applied Genetic Technologies Corporation, Alachua, Florida.
  • Shearman MS; Applied Genetic Technologies Corporation, Alachua, Florida.
  • Aguirre GD; Division of Experimental Retinal Therapies, Department of Clinical Sciences & Advanced Medicine, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.
  • Beltran WA; Division of Experimental Retinal Therapies, Department of Clinical Sciences & Advanced Medicine, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.
Hum Gene Ther ; 31(3-4): 253-267, 2020 02.
Article in En | MEDLINE | ID: mdl-31910043
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Genetic Therapy / Retinitis Pigmentosa / Dependovirus / Genes, X-Linked / Eye Proteins / Genetic Vectors Type of study: Diagnostic_studies / Prognostic_studies / Risk_factors_studies Limits: Animals / Humans Language: En Journal: Hum Gene Ther Journal subject: GENETICA MEDICA / TERAPEUTICA Year: 2020 Document type: Article Country of publication:
Fulltext
Add to My VHL
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Genetic Therapy / Retinitis Pigmentosa / Dependovirus / Genes, X-Linked / Eye Proteins / Genetic Vectors Type of study: Diagnostic_studies / Prognostic_studies / Risk_factors_studies Limits: Animals / Humans Language: En Journal: Hum Gene Ther Journal subject: GENETICA MEDICA / TERAPEUTICA Year: 2020 Document type: Article Country of publication: