Emerging potential of transposons for gene therapy and generation of induced pluripotent stem cells Export

@article{citeulike:4678584, abstract = {Effective gene therapy requires robust delivery of the desired genes into the relevant target cells, long-term gene expression, and minimal risks of secondary effects. The development of efficient and safe nonviral vectors would greatly facilitate clinical gene therapy studies. However, nonviral gene transfer approaches typically result in only limited stable gene transfer efficiencies in most primary cells. The use of nonviral gene delivery approaches in conjunction with the latest generation transposon technology based on Sleeping Beauty (SB) or piggyBac transposons may potentially overcome some of these limitations. In particular, a large-scale genetic screen in mammalian cells yielded a novel hyperactive SB transposase, resulting in robust and stable gene marking in vivo after hematopoietic reconstitution with CD34+ hematopoietic stem/progenitor cells in mouse models. Moreover, the first-in-man clinical trial has recently been approved to use redirected T cells engineered with SB for gene therapy of B-cell lymphoma. Finally, induced pluripotent stem cells could be generated after genetic reprogramming with piggyBac transposons encoding reprogramming factors. These recent developments underscore the emerging potential of transposons in gene therapy applications and induced pluripotent stem generation for regenerative medicine. 10.1182/blood-2009-04-210427}, author = {Vandendriessche, Thierry and Ivics, Zoltan and Izsvak, Zsuzsanna and Chuah, Marinee K.}, citeulike-article-id = {4678584}, citeulike-linkout-0 = {http://dx.doi.org/10.1182/blood-2009-04-210427}, citeulike-linkout-1 = {http://bloodjournal.hematologylibrary.org/cgi/content/abstract/114/8/1461}, citeulike-linkout-2 = {http://view.ncbi.nlm.nih.gov/pubmed/19471016}, citeulike-linkout-3 = {http://www.hubmed.org/display.cgi?uids=19471016}, doi = {10.1182/blood-2009-04-210427}, journal = {Blood}, keywords = {gene-therapy, stem-cell, transposon}, month = {August}, number = {8}, pages = {1461--1468}, posted-at = {2009-05-29 15:13:19}, priority = {2}, title = {Emerging potential of transposons for gene therapy and generation of induced pluripotent stem cells}, url = {http://dx.doi.org/10.1182/blood-2009-04-210427}, volume = {114}, year = {2009} }

TY - JOUR ID - citeulike:4678584 L3 - citeulike-article-id:4678584 N2 - Effective gene therapy requires robust delivery of the desired genes into the relevant target cells, long-term gene expression, and minimal risks of secondary effects. The development of efficient and safe nonviral vectors would greatly facilitate clinical gene therapy studies. However, nonviral gene transfer approaches typically result in only limited stable gene transfer efficiencies in most primary cells. The use of nonviral gene delivery approaches in conjunction with the latest generation transposon technology based on Sleeping Beauty (SB) or piggyBac transposons may potentially overcome some of these limitations. In particular, a large-scale genetic screen in mammalian cells yielded a novel hyperactive SB transposase, resulting in robust and stable gene marking in vivo after hematopoietic reconstitution with CD34+ hematopoietic stem/progenitor cells in mouse models. Moreover, the first-in-man clinical trial has recently been approved to use redirected T cells engineered with SB for gene therapy of B-cell lymphoma. Finally, induced pluripotent stem cells could be generated after genetic reprogramming with piggyBac transposons encoding reprogramming factors. These recent developments underscore the emerging potential of transposons in gene therapy applications and induced pluripotent stem generation for regenerative medicine. 10.1182/blood-2009-04-210427 IS - 8 JF - Blood EP - 1468 TI - Emerging potential of transposons for gene therapy and generation of induced pluripotent stem cells VL - 114 SP - 1461 KW - gene-therapy KW - stem-cell KW - transposon AU - Vandendriessche, Thierry AU - Ivics, Zoltan AU - Izsvak, Zsuzsanna AU - Chuah, Marinee PY - 2009/08/20/ UR - http://dx.doi.org/10.1182/blood-2009-04-210427 ER -