IN UTERO GENE THERAPY FOR FETUSES

摘要

A process for effecting gene therapy in utero in a fetus, which comprises transducing fetal cells in vivo with at least one nucleic acid sequence encoding a therapeutic agent. The fetal cells may be transduced with a viral vector (such as a retroviral vector) which includes the nucleic acid sequence encoding the therapeutic agent. The viral vector may be contained in a viral supernatant which is administered to the fetus, or may be generated by a producer cell line which is administered to the fetus.