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Updated: Jul 28, 2025

Laser-scanning Photostimulation of Optogenetically Targeted Forebrain Circuits

Laser-scanning Photostimulation of Optogenetically Targeted Forebrain Circuits

Published on: December 27, 2013

A clinically viable approach to restoring visual function using optogenetic gene therapy.

Boyuan Yan^1,2, Suresh Viswanathan³, Scott E Brodie⁴

¹Department of Physiology and Biophysics, Weill Cornell Medicine, New York, NY 10065, USA.

Molecular Therapy. Methods & Clinical Development

|May 30, 2023

Summary

This summary is machine-generated.

Optogenetic gene therapy using Chronos shows promise for vision restoration in retinal diseases. Preclinical studies confirm efficacy and safety in animal models, supporting clinical trials for retinitis pigmentosa.

Keywords:

optogenetic gene therapy preclinical study retinal prosthesis retinitis pigmentosa vision restoration

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Injections of AAV Vectors for Optogenetics in Anesthetized and Awake Behaving Non-Human Primate Brain

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Published on: August 4, 2021

Area of Science:

Ophthalmology
Gene Therapy
Neuroscience

Background:

Retinal degenerative diseases, like retinitis pigmentosa (RP), cause vision loss.
Optogenetic gene therapy is a potential strategy for vision restoration.
Several clinical trials are investigating optogenetic approaches.

Purpose of the Study:

To present preclinical efficacy and safety data for the NCT04278131 clinical trial.
To evaluate an adeno-associated virus serotype 2 (AAV2) vector delivering the Chronos optogenetic protein.

Main Methods:

Efficacy was assessed in mice using dose-dependent electroretinograms (ERGs).
Safety was evaluated in rats, nonhuman primates, and mice.
Safety assessments included immunohistochemistry, cell counts, ERGs, and ocular toxicology assays.

Main Results:

Chronos-expressing vectors demonstrated efficacy across various doses and light intensities.
No adverse findings related to the test article were observed in anatomical or electrophysiological assays.
The gene therapy approach was well tolerated in all animal models.

Conclusions:

Preclinical data support the safety and efficacy of AAV2-Chronos for vision restoration.
This approach holds potential for treating patients with retinal degenerative diseases.
The findings encourage further clinical investigation in humans.