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A plain language summary of the phase 1/2 study of AAV5-hRKp.RPGR (botaretigene sparoparvovec) in people with RPGR-associated X-linked retinitis pigmentosa

Michaelides, M; Besirli, CG; Bainbridge, J; (2025) A plain language summary of the phase 1/2 study of AAV5-hRKp.RPGR (botaretigene sparoparvovec) in people with RPGR-associated X-linked retinitis pigmentosa. Future Rare Diseases , 5 (1) , Article 2535273. 10.1080/23995270.2025.2535273. Green open access

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Type: Article
Title: A plain language summary of the phase 1/2 study of AAV5-hRKp.RPGR (botaretigene sparoparvovec) in people with RPGR-associated X-linked retinitis pigmentosa
Open access status: An open access version is available from UCL Discovery
DOI: 10.1080/23995270.2025.2535273
Publisher version: https://doi.org/10.1080/23995270.2025.2535273
Language: English
Additional information: © 2025 The Author(s). Published by Informa UK Limited, trading as Taylor & Francis Group This is an Open Access article distributed under the terms of the Creative Commons Attribution-NonCommercial License (http://creativecommons.org/licenses/by-nc/4.0/), which permits unrestricted non-commercial use, distribution, and reproduction in any medium, provided the original work is properly cited. The terms on which this article has been published allow the posting of the Accepted Manuscript in a repository by the author(s) or with their consent.
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences > Institute of Ophthalmology
URI: https://discovery.ucl.ac.uk/id/eprint/10212309
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