...
  • 主题
高级搜索  >
历史搜索 清空历史
首页> 外文期刊>Journal of cellular biochemistry. >Adeno-associated virus-mediated gene transfer.
【24h】

Adeno-associated virus-mediated gene transfer.

机译：腺相关病毒介导的基因转移。

获取原文
获取原文并翻译 | 示例
           
页面导航
  • 摘要
  • 著录项
  • 相似文献
  • 相关主题

摘要

Although the remarkable versatility and efficacy of recombinant adeno-associated virus 2 (AAV2) vectors in transducing a wide variety of cells and tissues in vitro, and in numerous pre-clinical animal models of human diseases in vivo, have been well established, the published literature is replete with controversies with regard to the efficacy of AAV2 vectors in hematopoietic stem cell (HSC) transduction. A number of factors have contributed to these controversies, the molecular bases of which have begun to come to light in recent years. With the availability of several novel serotypes (AAV1 through AAV12), rational design of AAV capsid mutants, and strategies (self-complementary vector genomes, hematopoietic cell-specific promoters), it is indeed becoming feasible to achieve efficient transduction of HSC by AAV vectors. Using a murine serial bone marrow transplantation model in vivo, we have recently documented stable integration of the proviral AAV genome into mouse chromosomes, which does not lead to any overt hematological abnormalities. Thus, a better understanding of the AAV-HSC interactions, and the availability of a vast repertoire of novel serotype and capsid mutant vectors, are likely to have significant implications in the use of AAV vectors in high-efficiency transduction of HSCs as well as in gene therapy applications involving the hematopoietic system.
机译：尽管重组腺相关病毒2（AAV2）载体在体外转导多种细胞和组织以及体内许多人类疾病的临床前动物模型中具有显着的多功能性和功效，但该出版物关于AAV2载体在造血干细胞（HSC）转导中的功效，文献众多。引起这些争议的因素很多，近年来，其分子基础已经开始显现。随着几种新型血清型（AAV1至AAV12）的可用性，AAV衣壳突变体的合理设计和策略（自互补载体基因组，造血细胞特异性启动子），通过AAV载体实现HSC的有效转导确实变得可行。最近在体内使用鼠类串行骨髓移植模型，我们已经证明前病毒AAV基因组稳定整合到小鼠染色体中，不会导致任何明显的血液学异常。因此，对AAV-HSC相互作用的更好理解以及新型血清型和衣壳突变载体的广泛应用可能对AAV载体在HSC的高效转导以及在HSC的高效转导中的应用具有重要意义。基因治疗应用涉及造血系统。

著录项

相似文献

  • 外文文献
  • 中文文献
  • 专利
获取原文

客服邮箱：kefu@zhangqiaokeyan.com

京公网安备：11010802029741号 ICP备案号：京ICP备15016152号-6 六维联合信息科技 (北京) 有限公司©版权所有

  • 客服微信

  • 服务号