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Vision on gyrate atrophy

Onderzoeksgroep Verhaagen
Publicatiejaar 2024
Gepubliceerd in EMBO Molecular Medicine
Auteur(s) Arthur A Bergen, Mark Jn Buijs, Anneloor Lma Ten Asbroek, Berith M Balfoort, Camiel Jf Boon, J. Verhaagen, Marion M Brands, Ronald Ja Wanders, Clara Dm van Karnebeek, Riekelt H Houtkooper

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.

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