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Related Experiment Video

Updated: Jun 10, 2026

Limbal Approach-Subretinal Injection of Viral Vectors for Gene Therapy in Mice Retinal Pigment Epithelium
06:48

Limbal Approach-Subretinal Injection of Viral Vectors for Gene Therapy in Mice Retinal Pigment Epithelium

Published on: August 7, 2015

Gene therapy in the second eye of RPE65-deficient dogs improves retinal function.

M J Annear1, J T Bartoe, S E Barker

  • 1Department of Small Animal Clinical Sciences, Michigan State University, East Lansing, MI 48824, USA.

Gene Therapy
|August 13, 2010
PubMed
Summary

Immune responses did not hinder gene therapy for RPE65 gene rescue in dogs. Second eye treatment with recombinant adeno-associated viral vector (rAAV2) was effective despite prior treatment and antibody response.

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Area of Science:

  • Ophthalmology
  • Gene Therapy
  • Immunology

Background:

  • Leber congenital amaurosis type II (LCA II) is a severe inherited retinal dystrophy.
  • Gene therapy offers a potential treatment for genetic retinal diseases like LCA II.
  • RPE65 gene mutations are a common cause of LCA II.

Purpose of the Study:

  • To assess if pre-existing immune responses affect gene therapy efficacy.
  • To evaluate the safety and effectiveness of treating the second eye in RPE65-/- dogs previously treated in one eye.
  • To determine if serum neutralizing antibody (NAb) responses impact gene therapy outcomes.

Main Methods:

  • Bilateral subretinal injection of rAAV2.hRPE65p.hRPE65 in RPE65-/- dogs.
  • Second eye treatment occurred 85-180 days after the first eye treatment.

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Isolation, Culture, and Genetic Engineering of Mammalian Primary Pigment Epithelial Cells for Non-Viral Gene Therapy
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Last Updated: Jun 10, 2026

Limbal Approach-Subretinal Injection of Viral Vectors for Gene Therapy in Mice Retinal Pigment Epithelium
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  • Electroretinography (ERG) and vision testing were used to assess treatment efficacy.

    • Main Results:

    • Gene therapy successfully rescued vision in 16 of 18 treated eyes.
    • No significant difference in efficacy was observed between first and second treated eyes.
    • Serum NAb response to rAAV2 was detected in all animals but did not impede rescue in the second eye.

    Conclusions:

    • Prior gene therapy in one eye does not preclude successful rescue in the contralateral eye using rAAV2.
    • Immune responses, including NAb, did not prevent effective gene therapy delivery or function.
    • Findings support the potential for bilateral gene therapy in treating human LCA II patients.