OPUS 24 / NSC
Project title: A new approach for gene delivery into retinal bipolar cells.
This project aims to develop a novel proof-of-concept approach to therapeutic gene delivery using a modified Rabies virus as a vector specific to bipolar cells. We propose that specific targeting of the surviving cell population within the degenerated retina, especially bipolar cells (BC) by our modified Rabies virus (RV), pseudotyped with LRIT3 (RV-LRIT3), may constitute a new strategy of gene therapy in retinal diseases. We hypothesize that the proposed viral approach can reach many BCs thanks to the interactions with the TRPM1 channels and deliver high levels of light-gated opsins to restore vision. The main innovation of this project is using the RV, which can transfect up to 4 genes. Therefore, it can be used to deliver opsins with different excitation spectrums and increase overall light sensitivity. We suggest this technique will restore selective responses in the mice’s visual cortex and superior colliculus. We will utilize single-neuron recordings and behavioral visual discrimination tasks to estimate visual network selectivity in response to complex and moving stimuli.
The total funding of the project is 4 224 291,00 PLN.