REAGENT AND METHOD FOR REPAIRING FBN1^T7498C MUTATION USING BASE EDITING

摘要

A reagent and method for repairing FBN1^T7498C mutation using base editing. A kit for efficiently repairing FBN1^T7498C mutation is characterized by comprising a base editing system and a repair re-sgRNA for an FBN1^T7498C site. By using a base editing technique, FBN1^T7498C mutation can be repaired by means of an accurate CT single base mutation, thereby providing an efficient and safe method for treating Marfan syndrome caused by this type of mutation.