Vision on gyrate atrophy: why treat the eye?

Autor:	Bergen AA; Department of Human Genetics, Section Ophthalmogenetics, Amsterdam UMC, University of Amsterdam, 1105 AZ, Amsterdam, the Netherlands.; Department of Ophthalmology, Amsterdam UMC, University of Amsterdam, 1105 AZ, Amsterdam, the Netherlands.; Emma Center for Personalized Medicine, Amsterdam UMC, Amsterdam, the Netherlands., Buijs MJ; Department of Human Genetics, Section Ophthalmogenetics, Amsterdam UMC, University of Amsterdam, 1105 AZ, Amsterdam, the Netherlands., Ten Asbroek AL; Department of Human Genetics, Section Ophthalmogenetics, Amsterdam UMC, University of Amsterdam, 1105 AZ, Amsterdam, the Netherlands., Balfoort BM; Department of Paediatrics, Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, 1105 AZ, Amsterdam, the Netherlands.; Laboratory Genetic Metabolic Diseases, Amsterdam Gastroenterology, Endocrinology, and Metabolism, Amsterdam UMC, University of Amsterdam, 1105 AZ, Amsterdam, the Netherlands., Boon CJ; Department of Ophthalmology, Amsterdam UMC, University of Amsterdam, 1105 AZ, Amsterdam, the Netherlands.; Department of Ophthalmology, Leiden UMC, Leiden, the Netherlands., Brands MM; Emma Center for Personalized Medicine, Amsterdam UMC, Amsterdam, the Netherlands.; Department of Paediatrics, Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, 1105 AZ, Amsterdam, the Netherlands.; United for Metabolic Diseases, Amsterdam, The Netherlands., Wanders RJ; Laboratory Genetic Metabolic Diseases, Amsterdam Gastroenterology, Endocrinology, and Metabolism, Amsterdam UMC, University of Amsterdam, 1105 AZ, Amsterdam, the Netherlands.; United for Metabolic Diseases, Amsterdam, The Netherlands., van Karnebeek CD; Emma Center for Personalized Medicine, Amsterdam UMC, Amsterdam, the Netherlands.; Department of Paediatrics, Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, 1105 AZ, Amsterdam, the Netherlands.; United for Metabolic Diseases, Amsterdam, The Netherlands., Houtkooper RH; Emma Center for Personalized Medicine, Amsterdam UMC, Amsterdam, the Netherlands. r.h.houtkooper@amsterdamumc.nl.; Laboratory Genetic Metabolic Diseases, Amsterdam Gastroenterology, Endocrinology, and Metabolism, Amsterdam UMC, University of Amsterdam, 1105 AZ, Amsterdam, the Netherlands. r.h.houtkooper@amsterdamumc.nl.
Jazyk:	angličtina
Zdroj:	EMBO molecular medicine [EMBO Mol Med] 2024 Jan; Vol. 16 (1), pp. 4-7. Date of Electronic Publication: 2023 Dec 14.
DOI:	10.1038/s44321-023-00001-1
Abstrakt:	In the April issue of this Journal, Boffa and coworkers put forward a new therapeutic approach for Gyrate Atrophy of the Choroid and Retina (GACR; OMIM 258870) (Boffa et al, 2023). The authors propose to apply gene therapy to the liver for GACR, a metabolic disease primarily affecting eyesight due to retinal degeneration. Their vision is enthusiastically supported by a News and Views comment in the same issue (Seker Yilmaz and Gissen, 2023). However, based on disease pathology, patient's needs, ethical considerations, therapeutic developmental time lines, and current state of the art of gene therapy for liver and eye, we have a different view on this issue: We argue below that local treatment of the eye is the preferred option for GACR. (© 2023. The Author(s).)
Databáze:	MEDLINE
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