Optogenetic gene therapy for the treatment of retinitis pigmentosa: a systematic review of clinical trials
Summary
This systematic review evaluates clinical trials of optogenetic gene therapy for retinitis pigmentosa. The review covers trials using channelrhodopsin-based approaches delivered via viral vectors to surviving retinal ganglion cells or bipolar cells, restoring light sensitivity in degenerated retinas. Safety and preliminary efficacy data from early-phase human studies are synthesized.
Why it matters
Optogenetic gene therapy offers a potentially transformative approach to vision restoration that does not require implanted electronic devices. By rendering surviving inner retinal cells sensitive to light, this approach could treat a broad range of retinal degenerations. The review of early clinical trial data provides insight into the safety profile and visual outcomes of this first-in-class gene therapy for blindness.
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