This proposal from Dr. Chernoff aims to use Crispr/Cas9-based gene editing to achieve such repair in Schwann cells bearing three distinct types of NF2 mutations (large deletion, small deletion, point mutation) that are encountered in patients. We will then compare the ability of the mutant and repaired Schwann cells to transmit nerve signals normally and for their ability to divide and grow, with the expectation that repair of the mutations will also normalize the behavior of these cells.
On Giving Tuesday, we will raise funds to start this program, and 100% of your donations will go to this project, not used for any of the nonprofit operational costs!