发明名称:REAGENT AND METHOD FOR REPAIRING FBN1T7498C MUTATION USING BASE EDITING
摘要:A reagent and method for repairing FBN1T7498C mutation using base editing. A kit for efficiently repairing FBN1T7498C mutation is characterized by comprising a base editing system and a repair re-sgRNA for an FBN1T7498C site. By using a base editing technique, FBN1T7498C 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.