COMPOSITIONS AND METHODS FOR ACHIEVING HIGH LEVELS OF TRANSDUCTION IN HUMAN LIVER CELLS

摘要

Use of a rAAV3B vector to deliver gene products to human hepatocytes is described. The rAAV3B vectors achieve high levels of transduction even in the presence of pre-existing immunity to AAV8 or AAVrh10. Compositions and treatment regimens are described. Also provided are rAAV engineered to facilitate purification and methods of purifying the AAV.