Evidence map›Paper›PMID 37716450›Full record

ArticleAmerican journal of ophthalmology2024

Leber Hereditary Optic Neuropathy Gene Therapy: Longitudinal Relationships Among Visual Function and Anatomical Measures.

Byron L Lam, William J Feuer, Vittorio Porciatti, Janet L Davis, D Diane Zheng, Elizabeth A Vanner, Eleonore J Savatovsky, Diego E Alba, John Guy

Registry-linked trialOpen access · hybridAbstract readClinical Trial, Phase I
PubMed Full text at PMC Publisher
In one paragraph

Article in American journal of ophthalmology, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02161380 (An Open-label Dose Escalation Study of an Adeno-associated Virus Vector), which is not on this map. Cited by 8 papers.

0numbers the graph read from it
0cells of the map it votes in
8citing papers in PubMed
2.2field-weighted citation impact, top 12% 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.

NCT02161380 phase1completednot on this map

An Open-label Dose Escalation Study of an Adeno-associated Virus Vector (scAAV2-P1ND4v2) for Gene Therapy of Leber's Hereditary Optic Neuropathy (LHON) Caused by the G11778A Mutation in Mitochondrial DNA

TypeinterventionalSponsorByron LamRan2014 to 2025Enrolled28ConditionsLeber's Hereditary Optic NeuropathyArmsinjection of scAAV2-P1ND4v2 1.18x10e9 vg (Low),, injection of scAAV2-P1ND4v2 5.81 X10e9 vg (Med), injection of scAAV2-P1ND4v2 2.4 X10e10vg (High), injection of scAAV2-P1ND4v2 1.0 X10e11vg (Higher)
3 · Its place in the literature

Who cites it

8 citing papers in PubMed, 15 citations in OpenAlex.

  1. Trial
  2. Review
  3. Review
  4. Review
  5. Current clinical applications of AAV-mediated gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Review
  6. Review
  7. Recombinant adeno-associated virus as a delivery platform for ocular gene therapy: A comprehensive review.Molecular therapy : the journal of the American Society of Gene Therapy · 2024
    Review
  8. 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

9 authors at 1 institution in 1 country.

Byron L LamFrom the Bascom Palmer Eye Institute, University of Miami Miller School of Medicine, Miami, Florida, USA. Electronic address: blam@med.miami.edu.
William J FeuerFrom the Bascom Palmer Eye Institute, University of Miami Miller School of Medicine, Miami, Florida, USA.
Vittorio PorciattiFrom the Bascom Palmer Eye Institute, University of Miami Miller School of Medicine, Miami, Florida, USA.
Janet L DavisFrom the Bascom Palmer Eye Institute, University of Miami Miller School of Medicine, Miami, Florida, USA.
D Diane ZhengFrom the Bascom Palmer Eye Institute, University of Miami Miller School of Medicine, Miami, Florida, USA.
Elizabeth A VannerFrom the Bascom Palmer Eye Institute, University of Miami Miller School of Medicine, Miami, Florida, USA.
Eleonore J SavatovskyFrom the Bascom Palmer Eye Institute, University of Miami Miller School of Medicine, Miami, Florida, USA.
Diego E AlbaFrom the Bascom Palmer Eye Institute, University of Miami Miller School of Medicine, Miami, Florida, USA.
John GuyFrom the Bascom Palmer Eye Institute, University of Miami Miller School of Medicine, Miami, Florida, USA.
University of Miami · US

Funding

Shared Equipment ModuleP30EY014801 · NEI · UNIVERSITY OF MIAMI SCHOOL OF MEDICINE · PI Dmitry V Ivanov · 2004 to 2026
$12.2M
Leber's Hereditary Optic Neuropathy: Gene Therapy Clinical TrialU10EY023558 · NEI · UNIVERSITY OF MIAMI SCHOOL OF MEDICINE · PI GUY, JOHN · 2014 to 2018
$5.8M
Biostatistics Center for Leber's Hereditary Optic Neuropathy: Gene Therapy TrialU10EY024247 · NEI · UNIVERSITY OF MIAMI SCHOOL OF MEDICINE · PI FEUER, WILLIAM JACK · 2014 to 2018
$566k
Lebers Hereditary Optic Neuropathy: Gene TherapyUG1EY023558 · NEI · UNIVERSITY OF MIAMI SCHOOL OF MEDICINE · PI LAM, BYRON L · 2019 to 2022
$0k
NEI NIH HHS P30 EY014801NEI NIH HHS U10 EY023558NEI NIH HHS U10 EY024247NEI NIH HHS UG1 EY023558
6 · The paper itself

Abstract

purposeTo assess longitudinal relationships among visual function and anatomical measures of gene therapy in G11778A Leber hereditary optic neuropathy (LHON).

designPhase 1 clinical trial.

methodsThis was a single-institution study of patients with G11778A LHON. Patients with chronic bilateral visual loss >12 months (group 1, n = 11), acute bilateral visual loss <12 months (group 2, n = 9), or unilateral visual loss (group 3, n = 8) were administered unilateral intravitreal AAV2(Y444,500,730F)-P1ND4v2 injection with low, medium, high, and higher doses to worse eye for groups 1 and 2 and better eye for group 3. Oucome measures were best-corrected visual acuity (BCVA), visual field mean deviation (VF MD), steady-state pattern electroretinogram (SS-PERG), optical coherence tomography (OCT) retinal nerve fiber layer (RNFL) thickness and ganglion cell+inner plexiform layer (GCIPL) thickness, and National Eye Institute Visual Function Questionnaire (NEI-VFQ-25) scores. Mean follow-up was 33.6 months (range = 18-36 months).

resultsBaseline SS-PERG amplitude was much reduced in both eyes of all groups including asymptomatic eyes of group 3, and showed no appreciable changes irrespective of disease stage and treatment. Significant and progressive GCIPL and RNFL thinning occurred in all eyes; BCVA and VF MD fluctuated in treated and fellow eyes, with some eyes having modest improvement that may be related to natural history or to gene therapy. Mean NEI-VFQ-25 scores declined in group 3 subjects (P = .023),

conclusionAsymptomatic eyes in LHON patients with unilateral visual loss may be beyond the window of effective neuroprotection given reduced GCIPL and SS-PERG. Randomization of patients to an untreated control group would help to assess treatment effect by accounting for variable natural history. NOTE: Publication of this article is sponsored by the American Ophthalmological Society.

Indexed as

Optic Atrophy, Hereditary, LeberGenetic TherapyHumansRetinal Ganglion CellsTomography, Optical CoherenceVision DisordersVisual AcuityVisual Fields

Identifiers

PMID37716450
PMCPMC10842528
OpenAlexW4386790947

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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.