Evidence map›Paper›PMID 34040899›Full record

ArticleExpert opinion on orphan drugs2021

Is subretinal AAV gene replacement still the only viable treatment option for choroideremia?

Ruofan Connie Han, Lewis E Fry, Ariel Kantor, Michelle E McClements, Kanmin Xue, Robert E MacLaren

Open access · hybridAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Expert opinion on orphan drugs, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed
0.3field-weighted citation impact, top 45% 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

3 citing papers in PubMed, 5 citations in OpenAlex.

  1. Gene therapy for choroideremia: progress, potential and pitfalls.Expert opinion on biological therapy · 2025
    Review
  2. Choroideremia: Toward Regulatory Approval of Retinal Gene Therapy.Cold Spring Harbor perspectives in medicine · 2023
    Article
  3. 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

6 authors at 1 institution in 1 country.

Ruofan Connie HanNuffield Department of Clinical Neurosciences, University of Oxford, Oxford, UK.ORCID 0000-0002-5478-7684
Lewis E FryNuffield Department of Clinical Neurosciences, University of Oxford, Oxford, UK.
Ariel KantorNuffield Department of Clinical Neurosciences, University of Oxford, Oxford, UK.
Michelle E McClementsNuffield Department of Clinical Neurosciences, University of Oxford, Oxford, UK.ORCID 0000-0002-2941-4464
Kanmin XueNuffield Department of Clinical Neurosciences, University of Oxford, Oxford, UK.
Robert E MacLarenNuffield Department of Clinical Neurosciences, University of Oxford, Oxford, UK.
University of Oxford · GB

Funding

Wellcome Trust 216593
6 · The paper itself

Abstract

introductionChoroideremia is an X-linked inherited retinal degeneration resulting from mutations in the AREAS COVERED: The authors examine the pathophysiology and genetic basis of choroideremia. They summarize the status of ongoing gene therapy trials and discuss EXPERT OPINION: The authors conclude that AAV-mediated gene augmentation remains the most effective approach for choroideremia. Given the heterogeneity of

Indexed as

AAVantisense oligonucleotidesCHMChoroideremiaCRISPRgene therapynonsense suppressionREP1

Identifiers

PMID34040899
PMCPMC7610829
OpenAlexW3123138077

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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.