Evidence map›Paper›PMID 38177529›Full record

ArticleEMBO molecular medicine2024

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, Marion M Brands, Ronald Ja Wanders, Clara Dm van Karnebeek, Riekelt H Houtkooper

Open access · goldAbstract readLetter
In one paragraph

Article in EMBO molecular medicine, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.

0numbers the graph read from it
0cells of the map it votes in
5citing papers in PubMed
1.2field-weighted citation impact, top 20% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

5 citing papers in PubMed, 8 citations in OpenAlex.

  1. Article
  2. Review
  3. Article
  4. A cohort study of 19 patients with gyrate atrophy of the choroid and retina (GACR).Graefe's archive for clinical and experimental ophthalmology = Albrecht von Graefes Archiv fur klinische und experimentelle Ophthalmologie · 2024
    Article
  5. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

10 authors at 5 institutions in 1 country.

Arthur A BergenDepartment of Human Genetics, Section Ophthalmogenetics, Amsterdam UMC, University of Amsterdam, 1105 AZ, Amsterdam, the Netherlands.
Mark Jn BuijsDepartment of Human Genetics, Section Ophthalmogenetics, Amsterdam UMC, University of Amsterdam, 1105 AZ, Amsterdam, the Netherlands.
Anneloor Lma Ten AsbroekDepartment of Human Genetics, Section Ophthalmogenetics, Amsterdam UMC, University of Amsterdam, 1105 AZ, Amsterdam, the Netherlands.ORCID 0000-0001-7190-0784
Berith M BalfoortDepartment of Paediatrics, Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, 1105 AZ, Amsterdam, the Netherlands.ORCID 0000-0002-6999-044X
Camiel Jf BoonDepartment of Ophthalmology, Amsterdam UMC, University of Amsterdam, 1105 AZ, Amsterdam, the Netherlands.
Dutch GACR “Bird’s Eye View” Consortium
Marion M BrandsEmma Center for Personalized Medicine, Amsterdam UMC, Amsterdam, the Netherlands.
Ronald Ja WandersLaboratory Genetic Metabolic Diseases, Amsterdam Gastroenterology, Endocrinology, and Metabolism, Amsterdam UMC, University of Amsterdam, 1105 AZ, Amsterdam, the Netherlands.
Clara Dm van KarnebeekEmma Center for Personalized Medicine, Amsterdam UMC, Amsterdam, the Netherlands.
Riekelt H HoutkooperEmma Center for Personalized Medicine, Amsterdam UMC, Amsterdam, the Netherlands. r.h.houtkooper@amsterdamumc.nl.ORCID 0000-0001-9961-0842
Royal Netherlands Academy of Arts and Sciences · NLAmsterdam University Medical Centers · NLErasmus University Rotterdam · NLLeiden University · NLNetherlands Institute for Neuroscience · NL

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

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.

Indexed as

Gyrate AtrophyRetinal DegenerationAtrophyChoroidHumansRetina

Identifiers

PMID38177529
PMCPMC10883273
OpenAlexW4389953700

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.