Transposon-based vector systems for gene therapy clinical trials: challenges and considerations.

摘要: Much progress has been made in gene therapy, but significant challenges remain. One is development of a range different tools that can be used for therapeutic purposes. Another site-specific targeting safe and faithful expression. Viruses have long considered the most promising human therapy. However, fatal side effects associated with viral vectors hampered their clinical application. DNA transposons, widely utilized decades as genetic plants insects, are now emerging viable In this article, we will give brief review adverse virus-based therapy followed by glimpse adeno-associated virus vector system, which currently The transposon-based delivery systems advantages limits commonly transposon systems, Sleeping Beauty, Tol2, piggyBac, extensively discussed Finally, focus on system piggyBac. Challenges considerations advancing piggyBac application critically addressed.