Adeno-associated virus vector variant and method for highly efficient genome editing

摘要

Adeno-associated virus (AAV) Clade F vector or AAV vector variants and methods and kits for accurate editing of the cellular genome (compared to AAV9) are provided. Targeted genome editing using the AAV Clade F vector or AAV vector variant provided herein has been shown to be 1,000 to 100,000 times more efficient than previously reported. Occurred at a different frequency. Also, to treat the disease or disorder of the subject, the genome of the subject cell is edited by transducing the cell with the AAV Clade F vector or AAV vector variant described herein, and the transduction Also provided are methods of treating a disease or disorder in a subject by further transplanting the cells into the subject. Also provided herein is a method of treating a disease or disorder in a subject by in vivo genome editing by administering the AAV Clade F vector or AAV vector variant described herein directly to the subject.