Safety Considerations Associated with Development and Clinical Application of Lentiviral Vector Systems for Gene Transfer

Gary   L.   Buchschacher, Jr.

Abstract

Lentiviral vector systems continue to be developed and investigated for possible use in clinical gene transfer studies and for application as potential gene therapies. These vector systems, as a new technology and potentially novel medical therapy, raise a number of safety concerns, both real and perceived. Careful and thorough characterization of the lentiviral vector systems and of how they might interact with cellular genomes and other pathogens is necessary in order to maximize the safety of this technology as it is developed and the safety of both individuals undergoing gene transfer procedures and the general population. Identification of potential safety concerns should not prevent or unnecessarily delay the development of lentiviral vectors for use in clinical gene transfer, but suggest areas of investigation where it would be wise to allocate resources and effort.

Keywords: gene therapy, lentivirus, viral vector, safety, recombination, insertional mutagenesis, human endogenous retrovirus (herv), human immunodeficiency virus (hiv)