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Related Concept Videos

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Related Experiment Video

Updated: Jul 8, 2025

Limbal Approach-Subretinal Injection of Viral Vectors for Gene Therapy in Mice Retinal Pigment Epithelium
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Leber hereditary optic neuropathy gene therapy.

Byron L Lam1

  • 1Bascom Palmer Eye Institute, University of Miami Miller School of Medicine, Miami, Florida, USA.

Current Opinion in Ophthalmology
|December 20, 2023
PubMed
Summary

Unilateral gene therapy for Leber hereditary optic neuropathy (LHON) showed unexpected bilateral visual improvement, though overall efficacy remains limited. Future strategies focus on mitochondrial DNA delivery and base editing for better outcomes.

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Area of Science:

  • Ophthalmology
  • Genetics
  • Neurology

Background:

  • Leber hereditary optic neuropathy (LHON) is a maternally inherited mitochondrial disease.
  • LHON primarily affects young males, causing rapid, painless, bilateral vision loss.
  • Mutations in mitochondrial DNA (mtDNA) genes are responsible for LHON.

Conclusions:

  • Unilateral gene therapy led to unanticipated bilateral visual gains, possibly due to efficacy, natural history, or other factors.
  • Overall efficacy is limited, and spontaneous improvements complicate trial interpretation.
  • Future strategies include mitochondrially-targeted AAV and mitochondrial base editing for improved LHON treatment and prevention.