Evidence map›Paper›PMID 33601057›Full record

Trial reportMolecular therapy : the journal of the American Society of Gene Therapy2021

Immune function in X-linked retinoschisis subjects in an AAV8-RS1 phase I/IIa gene therapy trial.

Alaknanda Mishra, Camasamudram Vijayasarathy, Catherine A Cukras, Henry E Wiley, H Nida Sen, Yong Zeng, Lisa L Wei, Paul A Sieving

Registry-linked trialOpen access · bronzeAbstract readClinical Trial, Phase IClinical Trial, Phase II
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Molecular therapy : the journal of the American Society of Gene Therapy, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02317887 (A Phase I/IIa Study of RS1 Ocular Gene Transfer for X-linked Retinoschisis), which is not on this map. Cited by 23 papers, 3 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
23citing papers in PubMed, 3 pooled it
3.8field-weighted citation impact, top 6% 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.

NCT02317887 phase1 / phase2completednot on this map

A Phase I/IIa Study of RS1 Ocular Gene Transfer for X-linked Retinoschisis

TypeinterventionalSponsorVegaVect, Inc.Ran2015 to 2024Enrolled12ConditionsRetinoschisis, X-LinkedArmsRS1 AAV Vector
3 · Its place in the literature

Who cites it

23 citing papers in PubMed, 3 syntheses or guidelines pooled it, 36 citations in OpenAlex.

  1. A systematic review of immunosuppressive protocols used in AAV gene therapy for monogenic disorders.Molecular therapy : the journal of the American Society of Gene Therapy · 2024
    Pooled it
  2. Pooled it
  3. Pooled it
  4. Article
  5. Article
  6. Article
  7. Review
  8. Review
  9. Article
  10. Article
  11. Review
  12. Review
  13. Review
  14. Immunology of Retinitis Pigmentosa and Gene Therapy-Associated Uveitis.Cold Spring Harbor perspectives in medicine · 2024
    Review
  15. Cell-cell interaction in the pathogenesis of inherited retinal diseases.Frontiers in cell and developmental biology · 2024
    Review
  16. Article
  17. Article
  18. Review
  19. Article
  20. Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

8 authors at 1 institution in 1 country.

Alaknanda MishraNational Eye Institute, National Institutes of Health, Bethesda, MD 20892, USA.
Camasamudram VijayasarathyNational Eye Institute, National Institutes of Health, Bethesda, MD 20892, USA.
Catherine A CukrasNational Eye Institute, National Institutes of Health, Bethesda, MD 20892, USA.
Henry E WileyNational Eye Institute, National Institutes of Health, Bethesda, MD 20892, USA.
H Nida SenNational Eye Institute, National Institutes of Health, Bethesda, MD 20892, USA.
Yong ZengNational Eye Institute, National Institutes of Health, Bethesda, MD 20892, USA.
Lisa L WeiNational Eye Institute, National Institutes of Health, Bethesda, MD 20892, USA.
Paul A SievingNational Eye Institute, National Institutes of Health, Bethesda, MD 20892, USA; Department of Ophthalmology, University of California Davis, Davis, CA 95817, USA. Electronic address: pasieving@ucdavis.edu.
National Institutes of Health · US

Funding

Retinal Degeneration--genetic/environmental animal modelZ01DC000065 · NIDCD · NATIONAL INSTITUTE ON DEAFNESS AND OTHER COMMUNICATION DISORDERS · PI SIEVING, PAUL · 2002 to 2008
$2.5M
CELLULAR AND MOLECULAR NEUROSCIENCE OF SURGERY SYSTEMST32DC000065 · NIDCD · UNIVERSITY OF KENTUCKY · PI GETCHELL, THOMAS V · 2001 to 2005
$711k
A Phase I/IIa Study of RS1 Ocular Gene Transfer for X-linked RetinoschisisZIAEY000572 · NEI · NATIONAL EYE INSTITUTE · PI CHEW, EMILY Y · 2020 to 2025
$319k
Intramural NIH HHS Z01 DC000065NIDCD NIH HHS T32 DC000065
6 · The paper itself

Abstract

This study explored systemic immune changes in 11 subjects with X-linked retinoschisis (XLRS) in a phase I/IIa adeno-associated virus 8 (AAV8)-RS1 gene therapy trial (ClinicalTrials.gov: NCT02317887). Immune cell proportions and serum analytes were compared to 12 healthy male controls. At pre-dosing baseline the mean CD4/CD8 ratio of XLRS subjects was elevated. CD11c

Indexed as

Genetic TherapyCytokinesDependovirusDisease ManagementEye ProteinsGenetic Diseases, X-LinkedGenetic Predisposition to DiseaseGenetic VectorsHumansImmunityImmunity, CellularRetinoschisisT-Lymphocyte SubsetsTreatment OutcomeCytokinesEye ProteinsRS1 protein, humanAAV8 vectorcytokinesgene therapy clinical trialimmune functionocular inflammationretinoschisinT cellsX-linked retinoschisis

Identifiers

PMID33601057
PMCPMC8178519
OpenAlexW3132825024

What Socratic holds

Textmetadata
Read underepoch 390

Registered trials

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 Socratic graph.