Gene-therapy advances using canine and feline models of inherited retinal degeneration

Over 20 preclinical studies show adeno-associated viral (AAV) vectors restore retinal function in dogs and cats with single-gene inherited retinal degeneration, paralleling human disease. Veterinary gene-therapy trials directly inform human clinical protocols, confirming dogs and cats as essential translational models for ophthalmic research.

Journal: Eye
Sample Size: Review of multiple preclinical studies
Study Type: Narrative review
Published: 2025
Species:

Key Findings

  • Many inherited retinal degenerations in dogs and cats result from single-gene defects analogous to human diseases
  • Adeno-associated viral (AAV) vectors can deliver healthy gene copies to rescue retinal function
  • Successful veterinary trials have informed human clinical protocols
  • Dogs and cats serve as valuable translational models for ophthalmic gene therapy

The problem

The problem is that dogs and cats with inherited retinal degeneration often lose their vision because of single-gene defects. These conditions have no cure and lead to gradual blindness, sometimes starting in young animals. Until recently, there was no way to restore vision or halt the disease’s progress. Owners face the reality of watching their pet’s eyesight decline with no medical option to reverse or slow the loss.

How common this is

Inherited retinal degeneration affects a substantial number of pet dogs and cats worldwide. Specific breeds are at higher risk, but any animal can carry a single-gene defect that causes vision loss. While exact prevalence numbers for pets are not reported, similar hereditary eye diseases occur in people at rates as high as one in four thousand. Many pet owners only discover the problem after their animal bumps into furniture or hesitates in dim light, often after the disease has already advanced. In some families, multiple pets may be affected across generations due to shared genetics.

What the research found

The study found that adeno-associated viral (AAV) vectors, which are delivery vehicles for genetic material, can transfer healthy copies of defective genes directly into the retina of affected dogs and cats. This means gene therapy can target the exact mutation causing blindness, rather than treating symptoms. Veterinary trials using these gene-augmentation therapies resulted in measurable improvements in retinal function. In plain terms, treated pets regained some ability to see light or navigate their environment. Successful outcomes in animals led to protocols that are now used in human clinical trials for similar diseases. Practically, this suggests your pet’s condition can inform treatments for people with inherited blindness. When your dog or cat participates in these studies, it directly supports advances in both veterinary and human medicine. This means the line between pet and human care for retinal disorders is narrowing.

Why this matters

At home

If your cat starts bumping into walls or your dog hesitates at steps, vision loss may be progressing. You want to keep your pet safe and confident in their own space. Gene therapy offers a path to restoring light sensitivity and navigation skills, which means fewer accidents and a better quality of life. For example, a cat that can see changes in lighting may return to using the window perch, instead of staying hidden. A dog regaining some sight could resume daily walks without fear. For you, this means less stress and more time enjoying routines you both love.

With your veterinarian

When you bring your pet for an eye exam and genetic testing, your veterinarian can identify if a single-gene defect is causing the vision problem. Knowing that gene therapy protocols exist, you can discuss referral to specialty centers or clinical trials. This matters because early action may open doors to new treatments. If your dog’s condition matches those studied, your vet can help you navigate eligibility and next steps. For families with multiple affected pets, this also means future litters can be screened and managed proactively.

What you can do

  • Schedule a veterinary ophthalmology exam at the first sign of vision changes—such as bumping into objects or hesitating in dim light.
  • Request genetic testing for inherited retinal diseases if your pet’s breed is at risk or symptoms appear before age five.
  • Ask your veterinarian about current gene therapy trials or specialty centers offering advanced care for retinal degeneration.
  • Keep your pet’s environment consistent—use night lights and avoid rearranging furniture to support safe navigation.
  • Track changes in your pet’s behavior using a daily journal or video, then share these with your vet during appointments.
  • Discuss options for screening future litters if your breeding animals carry known single-gene defects.
  • Stay informed about new gene therapy developments by subscribing to veterinary ophthalmology newsletters or joining breed-specific health forums.

When to act

  • At the first sign of vision loss—schedule a veterinary eye exam within two weeks.
  • When genetic testing confirms a single-gene retinal defect—ask about gene therapy referral immediately.
  • If your pet is part of a breed known for inherited retinal degeneration—start annual eye screening by age two.
  • After a diagnosis—review eligibility for clinical trials with your veterinarian within one month.
  • When planning a breeding—screen both parents for genetic defects before mating.

Study limitations

  • The review synthesizes data from multiple preclinical studies rather than direct clinical trials.
  • No specific numbers on pet prevalence or success rates were reported.
  • Clinical accessibility of gene therapy for pet owners remains limited in most regions.
  • Dogs and cats in studies had naturally occurring single-gene retinal defects.
  • What we don’t know yet: long-term outcomes for treated pets beyond early trial periods.

Bottom line

Gene therapy using AAV vectors can restore retinal function in dogs and cats with inherited blindness. Early genetic testing and veterinary collaboration help you access new treatment options for your pet.

Your action plan

Schedule a veterinary ophthalmology exam for your pet if you notice any changes in navigation or response to light. If your cat or dog is from a breed with a history of inherited blindness, ask your veterinarian about genetic testing and start annual screenings by age two. Keep a journal of your pet’s behavior, noting any new difficulties with stairs, furniture, or light changes, and bring this record to your appointments. If genetic testing confirms a single-gene defect, discuss current gene therapy trials and specialty referral options right away. For families with breeding animals, arrange screening for both parents before planning a litter to reduce risk. Adjust your home environment with night lights and consistent layouts to keep your pet safe while you pursue treatment. Stay updated on new therapies by joining breed health forums and subscribing to veterinary newsletters.

Disclaimer

This brief is for educational purposes only and does not substitute for professional veterinary advice. Always consult your veterinarian before making decisions about your pet’s health or treatment options.

Reference

Multiple researchers. "Gene-therapy advances using canine and feline models of inherited retinal degeneration". Eye. 2025. DOI: 10.1038/s41433-025-03825-y