Fb1t7479c mutation repair kit, fb1t7489c mutation preparation and repair method, and method for repair of fb1t7489c mutation by base editing

摘要

The present invention provides reagents and methods for repairing FBN1T7498C mutations by base editing. The kit for efficiently repairing the FBN1T7498C mutation is characterized by containing a base editing system and a repair re-sgRNA for the FBN1T7498C site. The present invention provides an efficient and safe method for treating Marfan syndrome due to such mutations, which can repair the mutation of FBN1T7498C with an accurate CT single base mutation by base editing technology.