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Development of Methodology and Study Protocol: Safety and Efficacy of a Single Subretinal Injection of rAAV.hCNGA3 in Patients with CNGA3-Linked Achromatopsia Investigated in an Exploratory Dose-Escalation Trial.
Kahle, Nadine A; Peters, Tobias; Zobor, Ditta; Kuehlewein, Laura; Kohl, Susanne; Zhour, Ahmad; Werner, Annette; Seitz, Immanuel P; Sothilingam, Vithiyanjali; Michalakis, Stylianos; Biel, Martin; Ueffing, Marius; Zrenner, Eberhart; Bartz-Schmidt, Karl U; Fischer, M Dominik; Wilhelm, Barbara J C.
Affiliation
  • Kahle NA; 1 University Hospital Tuebingen , Centre for Ophthalmology, Tuebingen, Germany .
  • Peters T; 1 University Hospital Tuebingen , Centre for Ophthalmology, Tuebingen, Germany .
  • Zobor D; 1 University Hospital Tuebingen , Centre for Ophthalmology, Tuebingen, Germany .
  • Kuehlewein L; 1 University Hospital Tuebingen , Centre for Ophthalmology, Tuebingen, Germany .
  • Kohl S; 1 University Hospital Tuebingen , Centre for Ophthalmology, Tuebingen, Germany .
  • Zhour A; 1 University Hospital Tuebingen , Centre for Ophthalmology, Tuebingen, Germany .
  • Werner A; 1 University Hospital Tuebingen , Centre for Ophthalmology, Tuebingen, Germany .
  • Seitz IP; 1 University Hospital Tuebingen , Centre for Ophthalmology, Tuebingen, Germany .
  • Sothilingam V; 1 University Hospital Tuebingen , Centre for Ophthalmology, Tuebingen, Germany .
  • Michalakis S; 2 Center for Integrated Protein Science Munich CiPSM at the Department of Pharmacy-Center for Drug Research, Ludwig-Maximilians-Universität München , Munich, Germany .
  • Biel M; 2 Center for Integrated Protein Science Munich CiPSM at the Department of Pharmacy-Center for Drug Research, Ludwig-Maximilians-Universität München , Munich, Germany .
  • Ueffing M; 1 University Hospital Tuebingen , Centre for Ophthalmology, Tuebingen, Germany .
  • Zrenner E; 1 University Hospital Tuebingen , Centre for Ophthalmology, Tuebingen, Germany .
  • Bartz-Schmidt KU; 1 University Hospital Tuebingen , Centre for Ophthalmology, Tuebingen, Germany .
  • Fischer MD; 1 University Hospital Tuebingen , Centre for Ophthalmology, Tuebingen, Germany .
  • Wilhelm BJC; 3 STZ eyetrial at the Centre for Ophthalmology , Tuebingen, Germany .
Hum Gene Ther Clin Dev ; 29(3): 121-131, 2018 09.
Article in En | MEDLINE | ID: mdl-30187779
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Genetic Therapy / Color Vision Defects / Cyclic Nucleotide-Gated Cation Channels Limits: Adult / Aged / Female / Humans / Male / Middle aged Language: En Journal: Hum Gene Ther Clin Dev Year: 2018 Type: Article Affiliation country: Germany
Fulltext
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Genetic Therapy / Color Vision Defects / Cyclic Nucleotide-Gated Cation Channels Limits: Adult / Aged / Female / Humans / Male / Middle aged Language: En Journal: Hum Gene Ther Clin Dev Year: 2018 Type: Article Affiliation country: Germany