Lentiviral Vectors for Gene Therapy

Abstract Lentiviral vector (LV) originated from human immunodeficiency virus is the common gene transfer vector in gene therapy. With the development over the past two decades, the safety, specific targeting and transduction efficiency of LV have been improved continually. In terms of safety, the virus genome was split into several plasmids and their accessory gene elements were removed. Specific viral envelope protein and posttranscriptional regulatory elements were introduced in LV system for the tissues' specific targeting and transduction efficiency. Based on these improvements, LV has been used widely in gene therapy especially for treatments of haemoglobinopathies, B cell leukaemia, immunodeficiency diseases and neurodegenerative diseases. Key Concepts Lentiviral vector derived from the human immunodeficiency virus and its development needs the evaluation and selection of virus genome elements for safe and efficient gene transfer. Lentiviral vector has shown its efficiency and safety in many clinical trials and approved gene therapy drugs. For the transduction efficiency, specific target and safety, lentiviral vector is modified and improved continuously. Currently, scientists focus on the more effective promoter, specific envelope protein, and inducible and regulatable expression system. The random insertional mutagenesis and high production cost remain the challenges in the lentiviral vector application. Lentiviral vector performs well in haematological and immune system partial due to the ex vivo transduction and transfusion back into blood flow which circumvent the shortage like relative lower titre and insertional mutagenesis.