Zhuo-Hua Pan

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AAV dose-dependent transduction efficiency in retinal ganglion cells and functional efficacy of optogenetic vision restoration - Gene Therapy

Sep 4, 2024 | nature.com | Qi Lu |Zhuo-Hua Pan

AbstractOptogenetics is a promising approach for restoring vision to the blind after photoreceptor degeneration. The ability to restore vision through AAV-mediated delivery of light-sensitive proteins, especially channelrhodopsins, into retinal ganglion cells has been extensively demonstrated in animal models. For clinical application, knowledge of viral dose-dependent functional efficacy is desired.

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