Preclinical evaluation of optogenetic therapy by clinically relevant multi-luminance behavioral test

Optogenetic therapy holds great potential for treatment of late-stage photoreceptor degenerative diseases. However, clinical translation of such therapeutic candidates is usually impeded by inconsistency in evaluation of visual function between preclinical models and clinical practice. To address this gap, we have developed a Multi-Luminance Behavioural Test (MLBT) to measure visual function of preclinical pathogenic models. Specifically, murine models for photoreceptor degeneration were evaluated in a light-dark box under various illumination levels according to real-life situations. In this way, a threshold for illumination level with therapeutic efficacy could be determined for optogenetic therapy candidates. MLBT of preclinical animal models treated by optogenetic therapy allows objective evaluation of light sensitivity for different therapy candidates in a more clinically relevant situation. Translatability of MLBT in preclinical models for optogenetic therapy will be evaluated in upcoming clinical trials.

Yihan Ethan Wu (Ethan) is a high school student at Concordia Shanghai with strong interests in synthetic biology and biomedicine. He has gained prior biomedical research experience through summer internships at biotechnology companies, including UgeneX and SIMR Bio. In 2024, he received a national silver medal at HOSA while competing in the Health Education category. He was also awarded a silver medal at the iGEM 2024 Jamboree, where his team was nominated for Best Presentation for their project focused on upcycling PET plastic into beta-hydroxybutyrate using engineered microbial pathways.