Lentiviral vectors for gene therapy

Gene therapy holds great promise for the treatment of several inherited and acquired diseases in clinical medicine. However, despite substantial advances in gene delivery technology, the promise that gene therapy could have a significant role to play in developing therapeutic strategies has yet to be fully realised. The challenges facing gene therapists is to develop more effective gene delivery systems to overcome the current inadequacies of existing gene transfer systems, particularly in the area of direct in vivo delivery. An ideal vector would have the ability to integrate and sustain expression of the transgene while eliciting no immunological consequences attributed to the vector and without mediating adverse pathological consequences to the recipient. These features have been demonstrated for lentiviral vectors derived from lentiviruses such as Human Immunodeficiency Viruses. However, several concerns have been raised pertaining to the biosafety of vectors derived from a human pathogenic virus. Recently, much progress has been made in the design and functional analysis of lentiviral vectors and this, coupled to remarkable advances in biosafety, is rapidly making lentiviral vectors one of the most exciting and promising prospects for gene delivery.