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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.
Affiliation
  • 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 in En | MEDLINE | ID: mdl-28566226
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Full text: 1 Database: MEDLINE Main subject: Retina / Genetic Therapy / Carrier Proteins / Retinitis Pigmentosa / Genes, X-Linked / Eye Proteins / Mutation Type of study: Clinical_trials / Diagnostic_studies / Prognostic_studies Limits: Animals / Humans Language: En Journal: Mol Ther Journal subject: BIOLOGIA MOLECULAR / TERAPEUTICA Year: 2017 Type: Article
Fulltext
Add to My VHL
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Full text: 1 Database: MEDLINE Main subject: Retina / Genetic Therapy / Carrier Proteins / Retinitis Pigmentosa / Genes, X-Linked / Eye Proteins / Mutation Type of study: Clinical_trials / Diagnostic_studies / Prognostic_studies Limits: Animals / Humans Language: En Journal: Mol Ther Journal subject: BIOLOGIA MOLECULAR / TERAPEUTICA Year: 2017 Type: Article