id author title date pages extension mime words sentences flesch summary cache txt work_n4bpax2mgjbqxhnar53qnias2a Zoltán Ivics Nonviral Gene Delivery with the Sleeping Beauty Transposon System 2011 10 .pdf application/pdf 7471 724 55 Effective gene therapy requires robust delivery of therapeutic genes into relevant target cells, long-term gene to provide efficient stable gene transfer and sustained transgene expression in primary cell types, including ex vivo gene delivery procedure apparently did not compromise the potential of transposon-marked CD34 + cells to 2. Expression cassettes delivered by Sleeping Beauty transposon vectors. promoters by the transposon insertion, and simultaneously protect the expression of the therapeutic gene from position possible strategy, introducing an imposed bias into the insertion profile, ideally, targeted integration of the therapeutic gene into a ''safe'' site in the human genome, could Sleeping Beauty transposon-mediated gene therapy for differentiated from human embryonic stem cells following transposon-based gene delivery. transgene expression in primitive human CD34 + hematopoietic stem/progenitor cells, using the Sleeping Beauty transposon system. source of transposase for Sleeping Beauty-mediated gene insertion and expression in somatic cells and tissues. cells using transposon-mediated gene transfer. ./cache/work_n4bpax2mgjbqxhnar53qnias2a.pdf ./txt/work_n4bpax2mgjbqxhnar53qnias2a.txt