The possibility of editing RNA has profound therapeutic potential, but has remained largely theoretical. Focused investments by RSRT have already demonstrated the potential for correcting MECP2 mutations at the level of RNA. RSRT is currently increasing their investment to aggressively pursue this therapeutic approach.
Goals during the next three years are to improve specificity and efficiency of editing RNA in the brain and to identify optimal delivery methods.
*Used with the permission of RSRT
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