The Sleeping Beauty transposon system: a non-viral vector for gene therapy

被引：107
作者：
Aronovich, Elena L. [1 ]
McIvor, R. Scott [1 ]
Hackett, Perry B. [1 ]
机构：
[1] Univ Minnesota, Inst Human Genet, Dept Genet Cell Biol & Dev, Ctr Genome Engn, Minneapolis, MN 55455 USA
来源：
HUMAN MOLECULAR GENETICS | 2011年 / 20卷
关键词：
LONG-TERM EXPRESSION; PLASMID DNA; PHENOTYPIC CORRECTION; STEM-CELLS; TRANSGENE EXPRESSION; INDUCED PLURIPOTENT; HEMATOPOIETIC STEM; RETROVIRAL VECTOR; HEMOPHILIC MICE; PIG-LIVER;
D O I：
10.1093/hmg/ddr140
中图分类号：
Q5 [生物化学]; Q7 [分子生物学];
学科分类号：
071010 ; 081704 ;
摘要：
Over the past decade, the Sleeping Beauty (SB) transposon system has been developed as the leading non-viral vector for gene therapy. This vector combines the advantages of viruses and naked DNA. Here we review progress over the last 2 years in vector design, methods of delivery and safety that have supported its use in the clinic. Currently, the SB vector has been validated for ex vivo gene delivery to stem cells, including T-cells for the treatment of lymphoma. Progress in delivery of SB transposons to liver for treatment of various systemic diseases, such as hemophilia and mucopolysaccharidoses types I and VII, has encountered some problems, but even here progress is being made.
引用
收藏
页码：R14 / R20
页数：7
相关论文
共 92 条
[1]   Pig liver gene therapy by noninvasive interventionist catheterism [J].
Alino, S. F. ;
Herrero, M. J. ;
Noguera, I. ;
Dasi, F. ;
Sanchez, M. .
GENE THERAPY, 2007, 14 (04) :334-343
[2]  
ARONOVICH EL, IMMUNOSUPPRESS UNPUB
[3]   Protonged expression of a tysosomat enzyme in mouse tiver after Sleeping Beauty transposon-mediated gene detivery:: imptications for non-virat gene therapy of mucopotysaccharidoses [J].
Aronovich, Elena L. ;
Bell, Jason B. ;
Belur, Lalitha R. ;
Gunther, Roland ;
Koniar, Brenda ;
Erickson, David C. C. ;
Schachem, Patricia A. ;
Matise, Ilze ;
McIvor, R. Scott ;
Whitley, Chester B. ;
Hackett, Perry B. .
JOURNAL OF GENE MEDICINE, 2007, 9 (05) :403-415
[4]   Systemic Correction of Storage Disease in MPS I NOD/SCID Mice Using the Sleeping Beauty Transposon System [J].
Aronovich, Elena L. ;
Bell, Jason B. ;
Khan, Shaukat A. ;
Belur, Lalitha R. ;
Gunther, Roland ;
Koniar, Brenda ;
Schachern, Patricia A. ;
Parker, Josh B. ;
Carlson, Cathy S. ;
Whitley, Chester B. ;
McIvor, R. Scott ;
Gupta, Pankaj ;
Hackett, Perry B. .
MOLECULAR THERAPY, 2009, 17 (07) :1136-1144
[5]   Strategies to Modulate Immune Responses: A New Frontier for Gene Therapy [J].
Arruda, Valder R. ;
Favaro, Patricia ;
Finn, Jonathan D. .
MOLECULAR THERAPY, 2009, 17 (09) :1492-1503
[6]   Hyperactive transposase mutants of the Sleeping Beauty transposon [J].
Baus, J ;
Liu, L ;
Heggestad, AD ;
Sanz, S ;
Fletcher, BS .
MOLECULAR THERAPY, 2005, 12 (06) :1148-1156
[7]   Novel Hyperactive Transposons for Genetic Modification of Induced Pluripotent and Adult Stem Cells: A Nonviral Paradigm for Coaxed Differentiation [J].
Belay, Eyayu ;
Matrai, Janka ;
Acosta-Sanchez, Abel ;
Ma, Ling ;
Quattrocelli, Mattia ;
Mates, Lajos ;
Sancho-Bru, Pau ;
Geraerts, Martine ;
Yan, Bing ;
Vermeesch, Joris ;
Rincon, Melvin Yesid ;
Samara-Kuko, Ermira ;
Ivics, Zoltan ;
Verfaillie, Catherine ;
Sampaolesi, Maurilio ;
Izsvak, Zsuzsanna ;
VandenDriessche, Thierry ;
Chuah, Marinee K. L. .
STEM CELLS, 2010, 28 (10) :1760-1771
[8]   Heme oxygenase-1 gene delivery by Sleeping Beauty inhibits vascular stasis in a murine model of sickle cell disease [J].
Belcher, John D. ;
Vineyard, Julie V. ;
Bruzzone, Carol M. ;
Chen, Chunsheng ;
Beckman, Joan D. ;
Nguyen, Julia ;
Steer, Clifford J. ;
Vercellotti, Gregory M. .
JOURNAL OF MOLECULAR MEDICINE-JMM, 2010, 88 (07) :665-675
[9]   Preferential delivery of the Sleeping Beauty transposon system to livers of mice by hydrodynamic injection [J].
Bell, Jason B. ;
Podetz-Pedersen, Kelly M. ;
Aronovich, Elena L. ;
Belur, Lalitha R. ;
McIvor, R. Scott ;
Hackett, Perry B. .
NATURE PROTOCOLS, 2007, 2 (12) :3153-3165
[10]   Duration of Expression and Activity of Sleeping Beauty Transposase in mouse liver following hydrodynamic DNA delivery [J].
Bell, Jason B. ;
Aronovich, Elena L. ;
Schreifels, Jeffrey M. ;
Beadnell, Thomas C. ;
Hackett, Perry B. .
MOLECULAR THERAPY, 2010, 18 (10) :1796-1802
12345678910