Protein Replacement
Protein replacement supplements MECP2 protein to the brain to make up for deficiency caused by the mutation of the MECP2 gene.
Protein Replacement Programs
UC Berkeley
RSRT is funding Alanna Schepartz, PhD, from UC Berkeley, who has discovered a family of proteins that are non-toxic and can deliver their cargo into the nucleus of cells with great efficiency. Importantly, the researchers have shown that the efficiency of transport can be determined with accuracy and precision.
The MECP2 protein is fused to the delivery protein and is then shuttled into individual cells in the brain. This approach has the potential to maximize the amount of protein that can be delivered to a cell and allows the dose to be tailored to the individual patient, allowing just the right amount to improve their symptoms.