Evidence map›Paper›PMID 31981492›Full record

ArticleMolecular therapy : the journal of the American Society of Gene Therapy2020

Gene Therapy for Glaucoma by Ciliary Body Aquaporin 1 Disruption Using CRISPR-Cas9.

Jiahui Wu, Oliver H Bell, David A Copland, Alison Young, John R Pooley, Ryea Maswood, Rachel S Evans, Peng Tee Khaw, Robin R Ali, Andrew D Dick and 1 more

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

Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 51 papers.

0numbers the graph read from it
0cells of the map it votes in
51citing papers in PubMed
4.6field-weighted citation impact, top 4% 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

51 citing papers in PubMed, 91 citations in OpenAlex.

  1. Article
  2. Review
  3. Review
  4. Review
  5. Review
  6. Article
  7. Review
  8. Article
  9. Review
  10. Article
  11. Review
  12. Review
  13. Genetic engineering and the eye.Eye (London, England) · 2025
    Review
  14. Review
  15. Review
  16. A Mini-Review on Gene Therapy in Glaucoma and Future Directions.International journal of molecular sciences · 2024
    Review
  17. Article
  18. Article
  19. Article
  20. Review
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

11 authors at 3 institutions in 2 countries.

Jiahui WuTranslational Health Sciences, University of Bristol, Bristol BS8 1TD, UK; Department of Ophthalmology, Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai 200080, China.
Oliver H BellTranslational Health Sciences, University of Bristol, Bristol BS8 1TD, UK.
David A CoplandTranslational Health Sciences, University of Bristol, Bristol BS8 1TD, UK.
Alison YoungTranslational Health Sciences, University of Bristol, Bristol BS8 1TD, UK.
John R PooleyTranslational Health Sciences, University of Bristol, Bristol BS8 1TD, UK.
Ryea MaswoodUCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK.
Rachel S EvansJohn van Geest Centre for Brain Repair, Department of Clinical Neurosciences, University of Cambridge, Cambridge CB2 0PY, UK.
Peng Tee KhawUCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK; NIHR Biomedical Research Centre for Ophthalmology at Moorfields Eye Hospital and UCL Institute of Ophthalmology, London EC1V 2PD, UK.
Robin R AliUCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK; NIHR Biomedical Research Centre for Ophthalmology at Moorfields Eye Hospital and UCL Institute of Ophthalmology, London EC1V 2PD, UK.
Andrew D DickTranslational Health Sciences, University of Bristol, Bristol BS8 1TD, UK; UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK; NIHR Biomedical Research Centre for Ophthalmology at Moorfields Eye Hospital and UCL Institute of Ophthalmology, London EC1V 2PD, UK.
Colin J ChuTranslational Health Sciences, University of Bristol, Bristol BS8 1TD, UK. Electronic address: colin.chu@bristol.ac.uk.
University of Bristol · GBUniversity College London · GBUniversity of Cambridge · GB

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Glaucoma is a common cause of blindness, yet current therapeutic options are imperfect. Clinical trials have invariably shown that reduction in intraocular pressure (IOP) regardless of disease subtype prevents visual loss. Reducing ciliary body aqueous humor production can lower IOP, and the adeno-associated virus ShH10 serotype was identified as able to transduce mouse ciliary body epithelium following intravitreal injection. Using ShH10 to deliver a single vector CRISPR-Cas9 system disrupting Aquaporin 1 resulted in reduced IOP in treated eyes (10.4 ± 2.4 mmHg) compared with control (13.2 ± 2.0 mmHg) or non-injected eyes (13.1 ± 2.8 mmHg; p < 0.001; n = 12). Editing in the aquaporin 1 gene could be detected in ciliary body, and no off-target increases in corneal or retinal thickness were identified. In experimental mouse models of corticosteroid and microbead-induced ocular hypertension, IOP could be reduced to prevent ganglion cell loss (32 ± 4 /mm

Indexed as

Gene EditingGenetic TherapyAnimalsAquaporin 1Base SequenceCiliary BodyCRISPR-Cas SystemsDependovirusGene ExpressionGene TargetingGenetic VectorsGlaucomaMiceRetinaTransduction, GeneticTransgenesAquaporin 1AAVAquaporinciliary bodyCRISPR-Cas9gene editingGlaucomaintraocular pressure

Identifiers

PMID31981492
PMCPMC7054720
OpenAlexW2999353727

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.