Evaluating Cell-permeant Miniature Proteins (CPMPs) as a Strategy For Delivering Functional MeCP2 Into Model Cells and Neurons
Alanna Schepartz, PhD | Yale University
Alanna Schepartz from Yale University is an award-winning professor who has a strong reputation in synthetic biology. One of the challenges in protein augmentation therapy is to get sufficient amount of protein into cells and then into the nucleus. Dr Schepartz has discovered a small protein which she calls a mini-protein that facilitates the uptake of the protein into the cell in a dose-dependent manner. Schepartz proposes to fuse this mini-protein with MeCP2. This approach has the potential to maximize the amount of protein that can be delivered to a cell and allow us to tailor the dose of MeCP2 protein for each individual patient, allowing just the right amount to improve their symptoms.