Novel Strategies of Protecting RGCs and their Axons in NF1 OPG Models

The objective of this project is to build on the discoveries made in Phase A, with an increased emphasis on several potentially translatable strategies for neuroprotection and vision restoration in NF1-OPG by 1) RNAseq approaches to identify new targets for RGC protection in NF1-OPG mice, 2) testing the protective and regenerative effects of positive hits from the He lab’s CRISPR screen in NF1 OPG mice, 3) developing a gene therapy approach to restore Nf1-like GAP function in Nf1 mutant mice, and 4) developing myelination-promoting treatments for restoring vision in NF1-OPG mice.