Abstract
Despite the many promising therapeutic approaches identified in the laboratory, it has proven extremely challenging to translate basic science advances into the eye clinic. There are many recent examples of clinical trials (e.g., Holz FG, Sadda SR, Busbee B, JAMA Ophthalmology 136:666-677, 2018) failing at the most expensive phase three stage, unable to demonstrate efficacy in the patient population. As a community we must think carefully about how we select what goes into that pipeline. Translating vision restoration therapies from the bench to the bedside involves selecting the most appropriate animal models of retinal degeneration and then moving beyond morphology to deploy appropriate functional tests in vitro, in vivo, and in the clinic. In this review we summarize the functional assays available to researchers, future prospects, and highlight areas in need of further development.
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McGregor, J.E., Williams, D.R., Merigan, W.H. (2019). Functional Assessment of Vision Restoration. In: Bowes Rickman, C., Grimm, C., Anderson, R., Ash, J., LaVail, M., Hollyfield, J. (eds) Retinal Degenerative Diseases. Advances in Experimental Medicine and Biology, vol 1185. Springer, Cham. https://doi.org/10.1007/978-3-030-27378-1_24
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