Stealth gene therapy.

摘要

Just over 7 years ago, the first successful gene therapy for hemophilia appeared to be at hand, until an unexpected immune response against the vector capsid led to clearance of corrected liver cells and a loss of the replacement factor IX gene.2 Since that trial, much has been learned, and in 2011, it was reported that intravenous injection of an AAV8 vector encoding factor IX was able to achieve sustained factor IX expression in 6 individuals with hemophilia B.