A Step Toward Liver Gene Therapy: Efficient Correction of the Genetic Defect of Hepatocytes Isolated From a Patient With Crigler-Najjar Syndrome Type 1 With Lentiviral Vectors

Abstract

The SLIT protocol allowed for a high transduction of CN-1 hepatocytes and restoration of the expression of the deficient protein. Furthermore, long-term survival of lentivirally transduced CN-1 hepatocytes in the liver of immunodeficient mice was demonstrated. This study is therefore an important step toward human application of lentiviral gene therapy.