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AAV-mediated gene replacement therapy for LRAT-associated retinitis pigmentosa: a proof-of-concept study in a patient-based rat model.

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Title: AAV-mediated gene replacement therapy for LRAT-associated retinitis pigmentosa: a proof-of-concept study in a patient-based rat model.
Authors: El-Kalaani AM; Department of Ophthalmology, Amsterdam UMC, University of Amsterdam (UvA), Amsterdam, The Netherlands.; Department of Human Genetics, Amsterdam UMC, University of Amsterdam (UvA), Amsterdam, The Netherlands., Ten Brink JB; Department of Human Genetics, Amsterdam UMC, University of Amsterdam (UvA), Amsterdam, The Netherlands., Boon CJF; Department of Ophthalmology, Amsterdam UMC, University of Amsterdam (UvA), Amsterdam, The Netherlands.; Department of Ophthalmology, Leiden University Medical Center (LUMC), Leiden, The Netherlands., Koster C; Department of Ophthalmology, Amsterdam UMC, University of Amsterdam (UvA), Amsterdam, The Netherlands. c.koster@amsterdamumc.nl.; Department of Human Genetics, Amsterdam UMC, University of Amsterdam (UvA), Amsterdam, The Netherlands. c.koster@amsterdamumc.nl.; Emma Center for Personalized Medicine, Amsterdam UMC, Amsterdam, The Netherlands. c.koster@amsterdamumc.nl.
Source: Gene therapy [Gene Ther] 2026 Mar 10. Date of Electronic Publication: 2026 Mar 10.
Publication Type: Journal Article
Journal Info: Publisher: Nature Publishing Group Country of Publication: England NLM ID: 9421525 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1476-5462 (Electronic) Linking ISSN: 09697128 NLM ISO Abbreviation: Gene Ther Subsets: MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:1476-5462
DOI:10.1038/s41434-026-00601-9