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Home / Equine Research Bank / Vet Ophthalmol / Cell and Gene Therapy in Equine Ocular Disease.
Veterinary ophthalmology2026; 29(2); e70151; doi: 10.1111/vop.70151

Cell and Gene Therapy in Equine Ocular Disease.

Abstract: Equine ocular disease is common and often challenging to treat using traditional methods. This has led to the development of new therapies. Like human medicine, veterinary medicine is adopting cellular and gene therapy as innovative approaches. Equine ocular disease is a particularly promising area for these techniques. Notably, immune-mediated diseases (such as immune-mediated keratitis and equine recurrent uveitis), ulcerative keratitis, and infectious ocular diseases are of interest. Several ocular gene therapy products are approved for use in humans, and more are currently being researched in veterinary medicine. In veterinary practice, cell therapy mainly involves multipotent stromal cells or mesenchymal stem cells (MSCs), which are also widely studied in human medicine. This review aims to summarize the status of cell and gene therapy in equine ocular disease and provide background on the principles behind these treatments, as well as insights from human medicine. Although many in vitro studies and case series exist, a significant research gap remains. Despite growing clinical use, there are limited controlled in vivo studies assessing their safety, routes of administration, or effectiveness.
© 2026 The Author(s). Veterinary Ophthalmology published by Wiley Periodicals LLC on behalf of American College of Veterinary Ophthalmologists.
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Publication Date: 2026-02-02 PubMed ID: 41623202PubMed Central: PMC12862700DOI: 10.1111/vop.70151Google Scholar: Lookup
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Summary

This research summary has been generated with artificial intelligence and may contain errors and omissions. Refer to the original study to confirm details provided. Submit correction.

Cell and gene therapies are emerging as promising approaches for treating equine ocular diseases, which are often difficult to manage with traditional treatments. This review article summarizes current advancements and knowledge gaps in the use of these innovative therapies in horses’ eye diseases, drawing parallels with human medicine.

Introduction to Equine Ocular Disease and Traditional Challenges

  • Equine ocular diseases are common and often difficult to treat effectively using conventional methods.
  • Diseases such as immune-mediated keratitis, equine recurrent uveitis, ulcerative keratitis, and infectious ocular conditions present treatment challenges.
  • The complexity of these diseases necessitates the exploration of novel therapeutic options beyond standard pharmaceutical or surgical interventions.

Emergence of Cell and Gene Therapy

  • Cell and gene therapy represent innovative medical approaches increasingly adopted from human medicine into veterinary fields.
  • These therapies aim to modify disease mechanisms at the molecular or cellular level, potentially offering longer-lasting and more targeted treatments.
  • In equine ocular diseases, such therapies could address immune system dysfunction, tissue repair, and infection control directly.

Cell Therapy Approaches

  • Cell therapy in equine ophthalmology predominantly involves multipotent stromal cells or mesenchymal stem cells (MSCs).
  • MSCs possess immunomodulatory, anti-inflammatory, and regenerative properties, making them suitable candidates for treating ocular inflammation and tissue damage.
  • These therapies have been extensively studied in vitro and in human clinical settings, providing a rationale for their application in horses.
  • Veterinary applications include treatments aimed at restoring corneal integrity, reducing inflammation, and promoting healing.

Gene Therapy Approaches

  • Gene therapy involves delivering genetic material to ocular cells to correct, replace, or regulate genes causing disease.
  • Several gene therapy products have been approved for human ocular diseases, demonstrating safety and efficacy.
  • Research is ongoing to adapt these strategies for veterinary use, targeting specific equine ocular conditions.
  • Gene therapy could provide solutions for inherited diseases or persistent infections by offering targeted control at the genetic level.

Current Status and Research Gaps

  • While numerous in vitro studies and case series document the potential benefits of cell and gene therapies, robust in vivo controlled trials in horses are limited.
  • There is insufficient data on safety profiles, optimal routes of administration, dosages, and long-term outcomes for these therapies in equine eyes.
  • Further research is crucial to establish standardized protocols, optimize treatment efficacy, and evaluate potential risks such as immune reactions or genetic off-target effects.
  • Bridging knowledge from human ophthalmology can inform veterinary research but species-specific variations must be considered.

Conclusion and Future Directions

  • Cell and gene therapy hold significant promise for revolutionizing the management of equine ocular diseases, with potential benefits including effective immune modulation and enhanced tissue repair.
  • Current clinical use is expanding but should be accompanied by rigorous scientific evaluation to ensure safety and efficacy.
  • Integration of multidisciplinary research involving molecular biology, veterinary medicine, and human clinical data is essential to advance these therapies.
  • Ongoing developments may lead to approved veterinary-specific cell and gene therapies, improving the quality of life for affected horses.

Cite This Article

APA
Young KAS, Schnabel LV, Gilger BC. (2026). Cell and Gene Therapy in Equine Ocular Disease. Vet Ophthalmol, 29(2), e70151. https://doi.org/10.1111/vop.70151

Publication

Veterinary ophthalmology
ISSN: 1463-5224
NlmUniqueID: 100887377
Country: England
Language: English
Volume: 29
Issue: 2
Pages: e70151
PII: e70151

Researcher Affiliations

Young, Kimberly A S
  • Department of Clinical Sciences, College of Veterinary Medicine, North Carolina State University, Raleigh, North Carolina, USA.
Schnabel, Lauren V
  • Department of Clinical Sciences, College of Veterinary Medicine, North Carolina State University, Raleigh, North Carolina, USA.
Gilger, Brian C
  • Department of Clinical Sciences, College of Veterinary Medicine, North Carolina State University, Raleigh, North Carolina, USA.

MeSH Terms

  • Animals
  • Horses
  • Horse Diseases / therapy
  • Genetic Therapy / veterinary
  • Eye Diseases / veterinary
  • Eye Diseases / therapy
  • Cell- and Tissue-Based Therapy / veterinary

Grant Funding

  • T32OD011130 / NIH HHS

Conflict of Interest Statement

Artificial Intelligence Statement: The authors have not used AI to generate any part of the manuscript.

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