Ex vivo hepatocyte gene therapy: increased biosafety protocol for transduction in suspension with lentiviral vectors and immediate transplantation (SLIT).

摘要

Hepatocyte ex vivo gene therapy is an interesting alternative to liver transplantation to treat metabolic liver diseases. Having applied our suspension with lentiviral vectors and immediate transplantation (SLIT) protocol (transduction in suspension with lentiviral vectors and immediate hepato-cytes transplantation) in macaques (1) and human cells, (2) we recently demonstrated its feasibility, efficacy, and biosafety. However, unfortunately, we detected a low, but present, systemic dissemination of the vector in some extrahepatic tissues in our macaques (i).