Clinical Validity of Functional Outcomes in Optogenetic Therapy for Advanced Retinitis Pigmentosa
DOI:
https://doi.org/10.5281/zenodo.23302595Keywords:
Retinitis pigmentosa, Optogenetics, Visual function, Clinical outcomes, Vision restorationAbstract
Optogenetic therapy represents a promising experimental strategy based on the introduction of light sensitive proteins into surviving retinal cells to enable responses to light stimulation. This study aimed to analyze the clinical validity of functional outcomes used to evaluate optogenetic therapy in patients with advanced retinitis pigmentosa. A scoping review of the literature was conducted using scientific databases, and five articles were selected from 78 publications analyzed. The selected studies addressed the therapeutic principles of optogenetics, functional assessment methods, initial clinical findings, and criteria used to measure treatment benefits. Measures such as object localization, visual discrimination, spatial orientation, mobility, and quality of life are more closely related to patients’ daily needs, although methodological heterogeneity, small sample sizes, and the lack of standardized assessment instruments remain important limitations. It is concluded that evaluating the therapeutic potential of optogenetics should integrate objective measures of visual function and clinically meaningful functional outcomes, allowing a more consistent interpretation of clinical benefits.
References
BORCHERT, G. A. et al. Functional methods for evaluating the efficacy of retinal optogenetic therapy for vision restoration. Frontiers in Neuroscience, v. 20, art. 1812539, 2026. DOI: https://doi.org/10.3389/fnins.2026.1812539.
HERCE-HAGIWARA, B. et al. The effectiveness and value of sonpiretigene isteparvovec for the treatment of advanced retinitis pigmentosa. Journal of Managed Care & Specialty Pharmacy, v. 31, n. 8, p. 835-840, ago. 2025. DOI: https://doi.org/10.18553/jmcp.2025.31.8.835.
LINDNER, M. et al. Optogenetics for visual restoration: from proof of principle to translational challenges. Progress in Retinal and Eye Research, v. 91, art. 101089, 2022. DOI: https://doi.org/10.1016/j.preteyeres.2022.101089.
ROSIN, B.; BANIN, E.; SAHEL, J. A. Current status of clinical trials design and outcomes in retinal gene therapy. Cold Spring Harbor Perspectives in Medicine, v. 14, n. 7, art. a041301, 2024. DOI: https://doi.org/10.1101/cshperspect.a041301.
SAHEL, J. A. et al. Partial recovery of visual function in a blind patient after optogenetic therapy. Nature Medicine, v. 27, p. 1223-1229, 2021. DOI: https://doi.org/10.1038/s41591-021-01351-4.
VERBAKEL, S. K. et al. Non-syndromic retinitis pigmentosa. Progress in Retinal and Eye Research, v. 66, p. 157-186, 2018. DOI: https://doi.org/10.1016/j.preteyeres.2018.03.005. and Translational
VINGOLO, E. M. et al. Optogenetics as a novel therapeutic approach for ocular disease. Journal of Clinical Ophthalmology, v. 3, n. 4, art. 21, 2025. DOI: https://doi.org/10.3390/jcto3040021.
Downloads
Published
How to Cite
Issue
Section
License
Copyright (c) 2026 Joyce Silva Alves
This work is licensed under a Creative Commons Attribution 4.0 International License.
Os artigos publicados na International Journal of Health and Surgical Research são licenciados sob a Creative Commons Attribution 4.0 International (CC BY 4.0).
Isso permite que outros distribuam, remixem, adaptem e criem a partir do trabalho publicado, para qualquer finalidade, incluindo comercial, desde que seja dado o devido crédito ao autor original e à revista.
Os autores mantêm os direitos autorais de seus trabalhos e concedem à revista o direito de primeira publicação.
