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BRIEF RESEARCH REPORT article

Front. Med.

Sec. Ophthalmology

This article is part of the Research TopicRetinopathy of prematurity: an update on pathophysiology, diagnostic methods and treatmentView all 7 articles

Efavirenz Treatment Improves Retinal Vaso-obliteration and Pathological Neovascularization in a Mouse Model of Retinopathy of Prematurity

Provisionally accepted
  • 1Meharry Medical College, Nashville, United States
  • 2Augusta University, Augusta, United States

The final, formatted version of the article will be published soon.

Objectives: Previous studies have shown the metabolic and regulatory significance of CYP46A1 in the adult retina; however, its role in the developing retina is unknown. Here, we evaluate CYP46A1 expression and the impact of its activation in the developing mouse retina under normal and pathological conditions. Methods: Seven-day-old (P7) C57BL/6J mice maintained in room air (controls) or subjected to oxygen-induced retinopathy (OIR) were treated with/without 20 mg/kg efavirenz (EFV), a CYP46A1 activator administered intraperitoneally from P7 to P17. Results: Retinal cross sections and flat mounts were prepared to study retinal vasculature morphology, Müller and microglia activation, and ganglion cell viability. EFV treatment significantly reduced pathological neovascularization and the size of avascular and hypoxic areas in OIR mice retinas. EFV treatment additionally limited reactive gliosis and microglia activation and improved retinal ganglion cell survival in OIR mice. Conclusions: The current study demonstrates the developmental regulation of CYP46A1 and the dysregulated expression and levels of the downstream metabolite 24-Hydroxycholesterol (24HC) in OIR mice. The study further suggests that EFV treatment (in part via CYP46A1 activation) may improve key pathological features associated with pathological neovascularization in OIR mice.

Keywords: cholesterol metabolism, CYP46A1, efavirenz, prematurity, retinopathy

Received: 13 Nov 2025; Accepted: 09 Feb 2026.

Copyright: © 2026 Bailey, Rudd Zhong Manis, Seth, Rajpurohit, Oxenrider, Martin, Jadeja and Thounaojam. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.

* Correspondence: Menaka Chanu Thounaojam

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