# Cell and Gene Therapy in Equine Ocular Disease

**Authors:** Kimberly A. S. Young, Lauren V. Schnabel, Brian C. Gilger

PMC · DOI: 10.1111/vop.70151 · Veterinary Ophthalmology · 2026-02-02

## TL;DR

This paper reviews the use of cell and gene therapy for treating eye diseases in horses, highlighting their potential and current research gaps.

## Contribution

The paper provides a comprehensive review of cell and gene therapy applications in equine ocular disease, emphasizing their emerging role in veterinary medicine.

## Key findings

- Cell and gene therapies are promising for treating immune-mediated and infectious equine ocular diseases.
- Multipotent stromal cells and mesenchymal stem cells are commonly used in veterinary cell therapy.
- There is a lack of controlled in vivo studies on the safety and effectiveness of these therapies in horses.

## 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.

## Full-text entities

- **Genes:** Plasminogen Activator Inhibitor-1 [NCBI Gene 100033931], CCL2 [NCBI Gene 100034136], IFN-gamma [NCBI Gene 100034181], Tgfb2 (transforming growth factor, beta 2) [NCBI Gene 21808] {aka Tgf-beta2, Tgfb-2}, cystatin C [NCBI Gene 100058494], IL-8 [NCBI Gene 100037400], Decorin [NCBI Gene 100033862], IL-1RA [NCBI Gene 100034236], CD4 [NCBI Gene 100052502], MMP 14 [NCBI Gene 100034170], beta defensin 2 [NCBI Gene 100629494], SDF-1 [NCBI Gene 100049872], TSG-6 [NCBI Gene 100034068], lipocalin 2 [NCBI Gene 100070310], Tlr4 (toll-like receptor 4) [NCBI Gene 21898] {aka Lps, Ly87, Ran/M1, Rasl2-8}, Smad7 [NCBI Gene 100033853], Tenascin-C [NCBI Gene 100049835], BMP7 [NCBI Gene 100050299], TGF-beta1 [NCBI Gene 100033900], CD25 [NCBI Gene 100070292], CXCR4 [NCBI Gene 100050974], TLR4 [NCBI Gene 100066890], mTOR [NCBI Gene 100051341], IL-6 [NCBI Gene 100034196], IL-10 [NCBI Gene 100034187], EQMHCB2 (MHC class I heavy chain) [NCBI Gene 100034210] {aka Eqca-1}, TLR-3 [NCBI Gene 100009703], Foxp3 [NCBI Gene 100052226]
- **Diseases:** tumorigenicity (MESH:D002471), ocular pain (MESH:D058447), Infectious Ocular Disease (MESH:D003141), corneal fibrosis (MESH:D005355), cancer (MESH:D009369), genetic disease (MESH:D030342), Toxicity (MESH:D064420), Ocular Disease (MESH:D005128), teratomas (MESH:D013724), vision loss (MESH:D014786), autoimmunity (MESH:D001327), KCS (MESH:C537021), Retinal Detachment (MESH:D012163), musculoskeletal disease (MESH:D009140), beta-thalassemia (MESH:D017086), immune dysregulation (OMIM:614878), corneal scarring (MESH:D065306), sickle cell anemia (MESH:D000755), pain (MESH:D010146), burn (MESH:D002056), blindness (MESH:D001766), immune-mediated diseases (MESH:C567355), tumorigenesis (MESH:D063646), Equine Ocular Disease (MESH:D006734), ulcerative keratitis (MESH:D003320), corneal (MESH:D003316), Inflammation (MESH:D007249), chronic (MESH:D002908), corneal opacity (MESH:D003318), epiphora (MESH:D007766), ERU (MESH:D014605), corneal wounds (MESH:D014947), IMMK (MESH:D007634), congenital and acquired diseases (MESH:D000163), disease (MESH:D004194)
- **Chemicals:** PEI (-), NO (MESH:D009614), PGE-2 (MESH:D015232), prostaglandin (MESH:D011453), pIC (MESH:D011066), polyinosinic, polycytidylic acid (MESH:D011070), steroids (MESH:D013256), AMP (MESH:D000249)
- **Species:** Staphylococcus aureus (species) [taxon 1280], Canis lupus familiaris (dog, subspecies) [taxon 9615], Homo sapiens (human, species) [taxon 9606], Oryctolagus cuniculus (domestic rabbit, species) [taxon 9986], Ovis aries (domestic sheep, species) [taxon 9940], Equus caballus (domestic horse, species) [taxon 9796], Rattus norvegicus (brown rat, species) [taxon 10116], Escherichia coli (E. coli, species) [taxon 562], Pseudomonas (RNA similarity group I, genus) [taxon 286], Mus musculus (house mouse, species) [taxon 10090]

## Full text

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## References

193 references — full list in the complete paper: https://tomesphere.com/paper/PMC12862700/full.md

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Source: https://tomesphere.com/paper/PMC12862700