Evidence map›Paper›PMID 25936984›Full record

Trial reportThe New England journal of medicine2015

Improvement and decline in vision with gene therapy in childhood blindness.

Samuel G Jacobson, Artur V Cideciyan, Alejandro J Roman, Alexander Sumaroka, Sharon B Schwartz, Elise Heon, William W Hauswirth

Registry-linked trialOpen access · bronzeAbstract readClinical TrialMulticenter Study
PubMed Full text at PMC Publisher
In one paragraph

Trial report in The New England journal of medicine, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT00481546 (Phase I Trial of Ocular Subretinal Injection of a Recombinant Adeno-Associated Virus), which is not on this map. Cited by 197 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
197citing papers in PubMed, 2 pooled it
26.5field-weighted citation impact, top 1% 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.

NCT00481546 phase1completednot on this map

Phase I Trial of Ocular Subretinal Injection of a Recombinant Adeno-Associated Virus (rAAV2-CBSB-hRPE65) Gene Vector to Patients With Retinal Disease Due to RPE65 Mutations (Clinical Trials of Gene Therapy for Leber Congenital Amaurosis)

TypeinterventionalSponsorUniversity of PennsylvaniaRan2007 to 2016Enrolled15ConditionsAmaurosis of Leber, Retinal DiseasesArmsrAAV2-CBSB-hRPE65
3 · Its place in the literature

Who cites it

197 citing papers in PubMed, 2 syntheses or guidelines pooled it, 373 citations in OpenAlex.

  1. Pooled it
  2. [Gene therapy as a treatment concept for inherited retinal diseases].Der Ophthalmologe : Zeitschrift der Deutschen Ophthalmologischen Gesellschaft · 2015
    Pooled it
  3. Trial
  4. Immune function in X-linked retinoschisis subjects in an AAV8-RS1 phase I/IIa gene therapy trial.Molecular therapy : the journal of the American Society of Gene Therapy · 2021
    Trial
  5. Trial
  6. Trial
  7. Trial
  8. Trial
  9. Article
  10. Article
  11. Article
  12. Review
  13. Article
  14. Review
  15. Article
  16. Review
  17. Novel photoreceptor-specific promoters for gene therapy in mid- to late-stage retinal degeneration.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Article
  18. Review
  19. Current clinical applications of AAV-mediated gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Review
  20. Canine models of inherited retinal diseases: from neglect to well-recognized translational value.Mammalian genome : official journal of the International Mammalian Genome Society · 2025
    Review

137 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

7 authors at 4 institutions in 3 countries.

Samuel G JacobsonFrom the Scheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia (S.G.J., A.V.C., A.J.R., A.S., S.B.S.); the Departments of Ophthalmology and Vision Sciences, the Hospital for Sick Children, University of Toronto, Toronto (E.H.); and the Department of Ophthalmology, University of Florida, Gainesville (W.W.H.).
Artur V Cideciyan
Alejandro J Roman
Alexander Sumaroka
Sharon B Schwartz
Elise Heon
William W Hauswirth
University of Pennsylvania · USPenn Presbyterian Medical Center · USHospital for Sick Children · CAUniversity of Florida · US

Funding

Scientific TransparencyP30EY001583 · NEI · UNIVERSITY OF PENNSYLVANIA · PI Qi N Cui · 1985 to 2026
$19.5M
Gene Therapy for Leber Congenital AmaurosisU10EY013729 · NEI · UNIVERSITY OF FLORIDA · PI HAUSWIRTH, WILLIAM W · 2001 to 2005
$11.5M
Clinical Trials of Gene Therapy for Leber Congenital AmaurosisU10EY017280 · NEI · UNIVERSITY OF PENNSYLVANIA · PI JACOBSON, SAMUEL GREGORY · 2006 to 2014
$9.8M
Vision Research CoreP30EY021721 · NEI · UNIVERSITY OF FLORIDA · PI HAUSWIRTH, WILLIAM W · 2011 to 2015
$2.8M
NEI NIH HHS EY013729NEI NIH HHS P30 EY001583NEI NIH HHS P30 EY021721NEI NIH HHS U10 EY013729NEI NIH HHS U10 EY017280
6 · The paper itself

Abstract

Retinal gene therapy for Leber's congenital amaurosis, an autosomal recessive childhood blindness, has been widely considered to be safe and efficacious. Three years after therapy, improvement in vision was maintained, but the rate of loss of photoreceptors in the treated retina was the same as that in the untreated retina. Here we describe long-term follow-up data from three treated patients. Topographic maps of visual sensitivity in treated regions, nearly 6 years after therapy for two of the patients and 4.5 years after therapy for the third patient, indicate progressive diminution of the areas of improved vision. (Funded by the National Eye Institute; ClinicalTrials.gov number, NCT00481546.).

Indexed as

Genetic TherapyAdolescentDisease ProgressionFollow-Up StudiesHumansLeber Congenital AmaurosisMutationPhotoreceptor Cells, VertebrateRetinaVision, OcularYoung Adult

Identifiers

PMID25936984
PMCPMC4450362
OpenAlexW2086498377

What OpenQuestion holds

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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 OpenQuestion graph.