Sleeping Beauty transposon vectors for therapeutic applications: advances and challenges

Transposable elements are natural, non-viral gene delivery vehicles capable of mediating stable genomic integration. The Sleeping Beauty (SB) transposon has the ability to cut-and-paste the 'gene of interest' into the genome, providing the basis for long-term, permanent transgene expression in transgenic cells and organisms. The SB transposon system is relatively well characterized, and has been extensively engineered for efficient gene delivery and gene discovery purposes in a wide range of vertebrates, including humans. The SB system is a safe and simple-to-use vector that enables cost-effective, rapid preparation of therapeutic doses of cell products. Recently, there has been a growing interest in using the SB system for therapy as evidenced by the large number of pre-clinical studies. SB moved swiftly from pre-clinical to clinical trials in almost a decade. In this article, we highlight the advancements and challenges associated with the SB system in various therapeutic applications. We also provide an overview that has been exploited by spin-off companies based on the SB system.