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基于睡美人转座子介导的基因疗法实现长期表达。

Sleeping beauty transposon-mediated gene therapy for prolonged expression.

作者信息

Hackett Perry B, Ekker Stephen C, Largaespada David A, McIvor R Scott

机构信息

Department of Genetics, Cell Biology and Development Arnold and Mabel Beckman Center for Transposon Research University of Minnesota Minneapolis, Minnesota 55455, USA.

出版信息

Adv Genet. 2005;54:189-232. doi: 10.1016/S0065-2660(05)54009-4.


DOI:10.1016/S0065-2660(05)54009-4
PMID:16096013
Abstract

The Sleeping Beauty (SB) transposon system represents a new vector for non-viral gene transfer that melds advantages of viruses and other forms of naked DNA transfer. The transposon itself is comprised of two inverted terminal repeats of about 340 base pairs each. The SB system directs precise transfer of specific constructs from a donor plasmid into a mammalian chromosome. The excision of the transposon from a donor plasmid and integration into a chromosomal site is mediated by Sleeping Beauty transposase, which can be delivered to cells vita its gene or its mRNA. As a result of its integration in chromosomes, and its lack of viral sequences that are often detected by poorly understood cellular defense mechanisms, a gene in a chromosomally integrated transposon can be expressed over the lifetime of a cell. SB transposons integrate nearly randomly into chromosomes at TA-dinucleotide base pairs although the sequences flanking the TAs can influence the probability of integration at a given site. Although random integration of vectors into human genomes is often thought to raise significant safety issues, evidence to date does not indicate that random insertions of SB transposons represent risks that are equal to those of viral vectors. Here we review the activities of the SB system in mice used as a model for human gene therapy, methods of delivery of the SB system, and its efficacy in ameliorating disorders that model human disease.

摘要

“睡美人”(SB)转座子系统是一种用于非病毒基因转移的新型载体,它融合了病毒和其他形式的裸DNA转移的优点。转座子本身由两个约340个碱基对的反向末端重复序列组成。SB系统可将特定构建体从供体质粒精确转移到哺乳动物染色体中。转座子从供体质粒上切除并整合到染色体位点是由“睡美人”转座酶介导的,转座酶可以通过其基因或mRNA传递到细胞中。由于其整合到染色体中,并且缺乏通常会被了解甚少的细胞防御机制检测到的病毒序列,因此染色体整合转座子中的基因可以在细胞的整个生命周期中表达。SB转座子几乎随机地整合到TA二核苷酸碱基对处的染色体中,尽管TA两侧的序列可以影响在给定位点整合的概率。尽管通常认为载体随机整合到人类基因组中会引发重大安全问题,但迄今为止的证据并未表明SB转座子的随机插入所带来的风险与病毒载体相当。在这里,我们综述了SB系统在用作人类基因治疗模型的小鼠中的活性、SB系统的递送方法及其在改善模拟人类疾病的病症方面的功效。

相似文献

[1]
Sleeping beauty transposon-mediated gene therapy for prolonged expression.

Adv Genet. 2005

[2]
Sleeping Beauty, a wide host-range transposon vector for genetic transformation in vertebrates.

J Mol Biol. 2000-9-8

[3]
Integration and long-term expression in xenografted human glioblastoma cells using a plasmid-based transposon system.

Mol Ther. 2004-8

[4]
Excision of Sleeping Beauty transposons: parameters and applications to gene therapy.

J Gene Med. 2004-5

[5]
Counterselection and co-delivery of transposon and transposase functions for Sleeping Beauty-mediated transposition in cultured mammalian cells.

Biosci Rep. 2004-12

[6]
Cis-acting gene regulatory activities in the terminal regions of sleeping beauty DNA transposon-based vectors.

Hum Gene Ther. 2007-12

[7]
Prolonged expression of a lysosomal enzyme in mouse liver after Sleeping Beauty transposon-mediated gene delivery: implications for non-viral gene therapy of mucopolysaccharidoses.

J Gene Med. 2007-5

[8]
Sleeping Beauty Transposition.

Microbiol Spectr. 2015-4

[9]
Messenger RNA as a source of transposase for sleeping beauty transposon-mediated correction of hereditary tyrosinemia type I.

Mol Ther. 2007-7

[10]
Stable transgene expression in primitive human CD34+ hematopoietic stem/progenitor cells, using the Sleeping Beauty transposon system.

Hum Gene Ther. 2009-12

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[3]
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[4]
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[5]
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[6]
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[7]
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[8]
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[9]
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[10]
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