Abstract

Summary

This review covers advances in ocular gene therapy for blindness, including gene replacement, neuroprotection, and optogenetic strategies that could restore light sensitivity to degenerated retinas. For lighting and circadian health applications, optogenetic approaches are particularly relevant as they may restore ipRGC-mediated responses to light, potentially recovering circadian entrainment capacity in blind individuals.
Abstract

Key Findings

  • Proof-of-concept studies were underway as of 2013 evaluating safety and efficacy of human retinal gene therapies, including optogenetic approaches
  • Optogenetic therapy represents a novel avenue for restoring vision by introducing light-sensitive proteins (opsins) into surviving retinal cells in degenerative conditions
  • The eye is identified as an optimal compartment for gene delivery due to its accessibility and immune privilege, supporting feasibility of clinical translation
Categories

Categories

Eye Health & Vision: Reviews gene therapy approaches for restoring vision in blinding retinal diseases, directly relevant to preserving photoreceptor function.
The Science of Light: Discusses optogenetic therapies that introduce light-sensitive proteins into retinal cells, relevant to photoreceptor biology and phototransduction.
Authors

Author(s)

JA Sahel, B Roska
Publication Date

Publication Year

2013
Citations

Number of Citations

176
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