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Retinal AAV8-RS1 Gene Therapy for X-Linked Retinoschisis: Initial Findings from a Phase I/IIa Trial by Intravitreal Delivery.
Cukras, Catherine; Wiley, Henry E; Jeffrey, Brett G; Sen, H Nida; Turriff, Amy; Zeng, Yong; Vijayasarathy, Camasamudram; Marangoni, Dario; Ziccardi, Lucia; Kjellstrom, Sten; Park, Tae Kwon; Hiriyanna, Suja; Wright, J Fraser; Colosi, Peter; Wu, Zhijian; Bush, Ronald A; Wei, Lisa L; Sieving, Paul A.
Affiliation
  • Cukras C; NIH, National Eye Institute, Bethesda, MD 20892, USA.
  • Wiley HE; NIH, National Eye Institute, Bethesda, MD 20892, USA.
  • Jeffrey BG; NIH, National Eye Institute, Bethesda, MD 20892, USA.
  • Sen HN; NIH, National Eye Institute, Bethesda, MD 20892, USA.
  • Turriff A; NIH, National Eye Institute, Bethesda, MD 20892, USA.
  • Zeng Y; STRRMD/NIDCD/NIH, Bethesda, MD 20874, USA.
  • Vijayasarathy C; STRRMD/NIDCD/NIH, Bethesda, MD 20874, USA.
  • Marangoni D; STRRMD/NIDCD/NIH, Bethesda, MD 20874, USA.
  • Ziccardi L; Neurophysiology of Vision and Neuropathmology Unit, IRCCS Fondazione G.B. Bietti, Rome 00198, Italy.
  • Kjellstrom S; Department of Ophthalmology, Lund University, Lund, Sweden.
  • Park TK; Ophthalomology, Soonchunhyang University Bucheon Hospital, Seoul, Korea.
  • Hiriyanna S; Ocular Gene Therapy Core, National Eye Institute, Bethesda, MD 20892, USA.
  • Wright JF; Ophthalomology, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Colosi P; Ocular Gene Therapy Core, National Eye Institute, Bethesda, MD 20892, USA.
  • Wu Z; NIH, National Eye Institute, Bethesda, MD 20892, USA.
  • Bush RA; STRRMD/NIDCD/NIH, Bethesda, MD 20874, USA.
  • Wei LL; NIH, National Eye Institute, Bethesda, MD 20892, USA.
  • Sieving PA; NIH, National Eye Institute, Bethesda, MD 20892, USA. Electronic address: paulsieving@nei.nih.gov.
Mol Ther ; 26(9): 2282-2294, 2018 09 05.
Article in En | MEDLINE | ID: mdl-30196853
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Genetic Therapy / Retinoschisis / Eye Proteins Type of study: Diagnostic_studies Limits: Adult / Aged / Female / Humans / Male / Middle aged Language: En Journal: Mol Ther Year: 2018 Document type: Article
Fulltext
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PubMed Links
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Genetic Therapy / Retinoschisis / Eye Proteins Type of study: Diagnostic_studies Limits: Adult / Aged / Female / Humans / Male / Middle aged Language: En Journal: Mol Ther Year: 2018 Document type: Article