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Gene therapy outcomes in young patients with RPE65-retinal degeneration.

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Title: Gene therapy outcomes in young patients with RPE65-retinal degeneration.
Authors: Stephenson KAJ; Department of Ophthalmology and Vision Sciences, The Hospital for Sick Children, Toronto, ON, Canada., Alsalamah AK; Department of Ophthalmology and Vision Sciences, The Hospital for Sick Children, Toronto, ON, Canada; Vitreoretinal Division, Ocular Genetics Service, King Khaled Eye Specialist Hospital, Riyadh, Saudi Arabia., Tumber A; Department of Ophthalmology and Vision Sciences, The Hospital for Sick Children, Toronto, ON, Canada., Mercer GD; Department of Ophthalmology and Vision Sciences, The Hospital for Sick Children, Toronto, ON, Canada; Unity Health St. Michael's Hospital, Toronto, ON, Canada; Department of Ophthalmology and Vision Sciences, University of Toronto, Toronto, ON, Canada., Ballios BG; Department of Ophthalmology and Vision Sciences, University of Toronto, Toronto, ON, Canada; University Health Network, Toronto, ON, Canada; Kensington Vision and Research Centre, Toronto, ON, Canada; Sunnybrook Health Sciences Centre, Toronto, ON, Canada., Muni RH; Department of Ophthalmology and Vision Sciences, The Hospital for Sick Children, Toronto, ON, Canada; Unity Health St. Michael's Hospital, Toronto, ON, Canada; Department of Ophthalmology and Vision Sciences, University of Toronto, Toronto, ON, Canada., Tavares E; Department of Ophthalmology and Vision Sciences, The Hospital for Sick Children, Toronto, ON, Canada; The Hospital for Sick Children, Programme of Genetics and Genome Biology, Toronto, ON, Canada., Kertes PJ; Department of Ophthalmology and Vision Sciences, The Hospital for Sick Children, Toronto, ON, Canada; Department of Ophthalmology and Vision Sciences, University of Toronto, Toronto, ON, Canada; University Health Network, Toronto, ON, Canada; Kensington Vision and Research Centre, Toronto, ON, Canada; Sunnybrook Health Sciences Centre, Toronto, ON, Canada., Vincent A; Department of Ophthalmology and Vision Sciences, The Hospital for Sick Children, Toronto, ON, Canada; Department of Ophthalmology and Vision Sciences, University of Toronto, Toronto, ON, Canada; The Hospital for Sick Children, Programme of Genetics and Genome Biology, Toronto, ON, Canada., Héon E; Department of Ophthalmology and Vision Sciences, The Hospital for Sick Children, Toronto, ON, Canada; Department of Ophthalmology and Vision Sciences, University of Toronto, Toronto, ON, Canada; The Hospital for Sick Children, Programme of Genetics and Genome Biology, Toronto, ON, Canada. Electronic address: elise.heon@sickkids.ca.
Source: Canadian journal of ophthalmology. Journal canadien d'ophtalmologie [Can J Ophthalmol] 2026 Jun; Vol. 61 (3), pp. 685-692. Date of Electronic Publication: 2026 Feb 27.
Publication Type: Journal Article
Journal Info: Publisher: Elsevier Country of Publication: England NLM ID: 0045312 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1715-3360 (Electronic) Linking ISSN: 00084182 NLM ISO Abbreviation: Can J Ophthalmol Subsets: MEDLINE
Database: MEDLINE Ultimate
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PubType: Academic Journal
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  Data: <searchLink fieldCode="AU" term="%22Stephenson+KAJ%22">Stephenson KAJ</searchLink>; Department of Ophthalmology and Vision Sciences, The Hospital for Sick Children, Toronto, ON, Canada.<br /><searchLink fieldCode="AU" term="%22Alsalamah+AK%22">Alsalamah AK</searchLink>; Department of Ophthalmology and Vision Sciences, The Hospital for Sick Children, Toronto, ON, Canada; Vitreoretinal Division, Ocular Genetics Service, King Khaled Eye Specialist Hospital, Riyadh, Saudi Arabia.<br /><searchLink fieldCode="AU" term="%22Tumber+A%22">Tumber A</searchLink>; Department of Ophthalmology and Vision Sciences, The Hospital for Sick Children, Toronto, ON, Canada.<br /><searchLink fieldCode="AU" term="%22Mercer+GD%22">Mercer GD</searchLink>; Department of Ophthalmology and Vision Sciences, The Hospital for Sick Children, Toronto, ON, Canada; Unity Health St. Michael's Hospital, Toronto, ON, Canada; Department of Ophthalmology and Vision Sciences, University of Toronto, Toronto, ON, Canada.<br /><searchLink fieldCode="AU" term="%22Ballios+BG%22">Ballios BG</searchLink>; Department of Ophthalmology and Vision Sciences, University of Toronto, Toronto, ON, Canada; University Health Network, Toronto, ON, Canada; Kensington Vision and Research Centre, Toronto, ON, Canada; Sunnybrook Health Sciences Centre, Toronto, ON, Canada.<br /><searchLink fieldCode="AU" term="%22Muni+RH%22">Muni RH</searchLink>; Department of Ophthalmology and Vision Sciences, The Hospital for Sick Children, Toronto, ON, Canada; Unity Health St. Michael's Hospital, Toronto, ON, Canada; Department of Ophthalmology and Vision Sciences, University of Toronto, Toronto, ON, Canada.<br /><searchLink fieldCode="AU" term="%22Tavares+E%22">Tavares E</searchLink>; Department of Ophthalmology and Vision Sciences, The Hospital for Sick Children, Toronto, ON, Canada; The Hospital for Sick Children, Programme of Genetics and Genome Biology, Toronto, ON, Canada.<br /><searchLink fieldCode="AU" term="%22Kertes+PJ%22">Kertes PJ</searchLink>; Department of Ophthalmology and Vision Sciences, The Hospital for Sick Children, Toronto, ON, Canada; Department of Ophthalmology and Vision Sciences, University of Toronto, Toronto, ON, Canada; University Health Network, Toronto, ON, Canada; Kensington Vision and Research Centre, Toronto, ON, Canada; Sunnybrook Health Sciences Centre, Toronto, ON, Canada.<br /><searchLink fieldCode="AU" term="%22Vincent+A%22">Vincent A</searchLink>; Department of Ophthalmology and Vision Sciences, The Hospital for Sick Children, Toronto, ON, Canada; Department of Ophthalmology and Vision Sciences, University of Toronto, Toronto, ON, Canada; The Hospital for Sick Children, Programme of Genetics and Genome Biology, Toronto, ON, Canada.<br /><searchLink fieldCode="AU" term="%22Héon+E%22">Héon E</searchLink>; Department of Ophthalmology and Vision Sciences, The Hospital for Sick Children, Toronto, ON, Canada; Department of Ophthalmology and Vision Sciences, University of Toronto, Toronto, ON, Canada; The Hospital for Sick Children, Programme of Genetics and Genome Biology, Toronto, ON, Canada. Electronic address: elise.heon@sickkids.ca.
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  Data: <searchLink fieldCode="JN" term="%220045312%22">Canadian journal of ophthalmology. Journal canadien d'ophtalmologie</searchLink> [Can J Ophthalmol] 2026 Jun; Vol. 61 (3), pp. 685-692. <i>Date of Electronic Publication: </i>2026 Feb 27.
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Elsevier%22">Elsevier </searchLink><i>Country of Publication: </i>England <i>NLM ID: </i>0045312 <i>Publication Model: </i>Print-Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>1715-3360 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2200084182%22">00084182 </searchLink><i>NLM ISO Abbreviation: </i>Can J Ophthalmol <i>Subsets: </i>MEDLINE
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      – Type: doi
        Value: 10.1016/j.jcjo.2026.01.016
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      – Code: eng
        Text: English
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      – TitleFull: Gene therapy outcomes in young patients with RPE65-retinal degeneration.
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              M: 06
              Text: 2026 Jun
              Type: published
              Y: 2026
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