Vision on gyrate atrophy: why treat the eye?

Arthur A. Bergen; Mark Jn Buijs; Anneloor Lma ten Asbroek; Berith M. Balfoort; Camiel Jf Boon; Dutch GACR “Bird’s Eye View” Consortium

doi:https://doi.org/10.1038/s44321-023-00001-1

Vision on gyrate atrophy: why treat the eye?

Arthur A. Bergen, Mark Jn Buijs, Anneloor Lma ten Asbroek, Berith M. Balfoort, Camiel Jf Boon, Dutch GACR “Bird’s Eye View” Consortium

Research output: Contribution to journal › Comment/Letter to the editor › Academic

Abstract

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.

Original language	English
Pages (from-to)	4-7
Number of pages	4
Journal	EMBO molecular medicine
Volume	16
Issue number	1
DOIs	https://doi.org/10.1038/s44321-023-00001-1
Publication status	Published - 16 Jan 2024

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title = "Vision on gyrate atrophy: why treat the eye?",

abstract = "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.",

author = "Bergen, {Arthur A.} and Buijs, {Mark Jn} and {ten Asbroek}, {Anneloor Lma} and Balfoort, {Berith M.} and Boon, {Camiel Jf} and {Dutch GACR “Bird{\textquoteright}s Eye View” Consortium} and Brands, {Marion M.} and Wanders, {Ronald Ja} and {van Karnebeek}, {Clara Dm} and Houtkooper, {Riekelt H.}",

note = "Publisher Copyright: {\textcopyright} The Author(s) 2023.",

year = "2024",

month = jan,

day = "16",

doi = "https://doi.org/10.1038/s44321-023-00001-1",

language = "English",

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T1 - Vision on gyrate atrophy

T2 - why treat the eye?

AU - Bergen, Arthur A.

AU - Buijs, Mark Jn

AU - ten Asbroek, Anneloor Lma

AU - Balfoort, Berith M.

AU - Boon, Camiel Jf

AU - Dutch GACR “Bird’s Eye View” Consortium

AU - Brands, Marion M.

AU - Wanders, Ronald Ja

AU - van Karnebeek, Clara Dm

AU - Houtkooper, Riekelt H.

PY - 2024/1/16

Y1 - 2024/1/16

N2 - 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.

AB - 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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U2 - https://doi.org/10.1038/s44321-023-00001-1

DO - https://doi.org/10.1038/s44321-023-00001-1

M3 - Comment/Letter to the editor

C2 - 38177529

SN - 1757-4676

VL - 16

SP - 4

EP - 7

JO - EMBO molecular medicine

JF - EMBO molecular medicine

IS - 1

ER -

Vision on gyrate atrophy: why treat the eye?

Abstract

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