Evidence map›Paper›PMID 41532047›Full record

ReviewJournal of medicine and life2025

Ocular gene therapy as a sustained drug delivery system: pharmacokinetic and genokinetic perspectives.

Carmen-Ecaterina Leferman, Alin Dumitru Ciubotaru

Abstract readReview
In one paragraph

Review in Journal of medicine and life, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed
–field-weighted citation impact
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

2 citing papers in PubMed.

  1. Article
  2. 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

2 authors.

Carmen-Ecaterina LefermanDepartment of Pharmacology, Grigore T. Popa University of Medicine and Pharmacy, Iasi, Romania.
Alin Dumitru CiubotaruDepartment of Biochemistry, Grigore T. Popa University of Medicine and Pharmacy, Iasi, Romania.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Ocular pharmacotherapy is constrained by compartmental anatomy and clearance barriers that limit sustained posterior-segment exposure. Intravitreal bolus dosing, therefore, remains dominant for retinal disease but produces peak-trough profiles and frequent retreatment. Long-acting implants and refillable systems can prolong exposure, yet are finite or maintenance-dependent. Ocular gene therapy introduces a different paradigm in which transduced retinal cells act as localized 'biofactories,' enabling prolonged intraocular production of therapeutic proteins after a single or infrequent administration. This review integrates pharmacokinetic principles with determinants of transgene expression, including vector/capsid design, promoter architecture, route-dependent biodistribution (subretinal, intravitreal, suprachoroidal), and immune modulation, to explain typical kinetics (lag phase, rise to plateau, and potential attenuation). We highlight an infusion-equivalent modeling framework that treats transgene-driven protein output as sustained input balanced by first-order loss, providing parameters for time to plateau, steady-state exposure, and variability. Finally, we discuss translational implications for efficacy and safety, including exposure-response and therapeutic window definition in emerging retinal gene therapy programs (notably anti-VEGF), and future directions such as tunable expression systems and biomarker-linked, model-informed dose optimization.

Indexed as

Drug Delivery SystemsGenetic TherapyRetinal DiseasesAnimalsGene Therapy AgentsHumansadeno-associated virusanti-VEGF therapyocular gene therapypharmacokineticsretinal diseasessustained drug delivery

Identifiers

PMID41532047
PMCPMC12794103

What Socratic holds

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LicenceCC BY
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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 Socratic graph.