【共享】好文推荐Engineering targeted viral vectors for gene therapy
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Nature Reviews Genetics 8, 573-587 (August 2007) | doi:10.1038/nrg2141
Engineering targeted viral vectors for gene therapy
Reinhard Waehler1, Stephen J. Russell2 & David T. Curiel1 About the authors
Top of page
Abstract
To achieve therapeutic success, transfer vehicles for gene therapy must be capable of transducing target cells while avoiding impact on non-target cells. Despite the high transduction efficiency of viral vectors, their tropism frequently does not match the therapeutic need. In the past, this lack of appropriate targeting allowed only partial exploitation of the great potential of gene therapy. Substantial progress in modifying viral vectors using diverse techniques now allows targeting to many cell types in vitro. Although important challenges remain for in vivo applications, the first clinical trials with targeted vectors have already begun to take place.
基因治疗的转运载体必须具备转化目的细胞的能力,同时又要避免作用于非靶细胞,这是治疗成功的必要条件.尽管病毒载体的转化效率很高,他们的靶向性却往往无法满足治疗要求.过去这种靶向性的缺乏使得基因治疗的巨大潜力仅仅得到部分开发.最近通过各种技术对病毒载体改造的稳步进展使得这些载体在体外对许多细胞的靶性作用成为可能.虽然仍然存在诸多挑战,第一个利用靶向性载体的临床实验已经开始进行了.
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Nature Reviews Genetics 8, 573-587 (August 2007) | doi:10.1038/nrg2141
Engineering targeted viral vectors for gene therapy
Reinhard Waehler1, Stephen J. Russell2 & David T. Curiel1 About the authors
Top of page
Abstract
To achieve therapeutic success, transfer vehicles for gene therapy must be capable of transducing target cells while avoiding impact on non-target cells. Despite the high transduction efficiency of viral vectors, their tropism frequently does not match the therapeutic need. In the past, this lack of appropriate targeting allowed only partial exploitation of the great potential of gene therapy. Substantial progress in modifying viral vectors using diverse techniques now allows targeting to many cell types in vitro. Although important challenges remain for in vivo applications, the first clinical trials with targeted vectors have already begun to take place.
基因治疗的转运载体必须具备转化目的细胞的能力,同时又要避免作用于非靶细胞,这是治疗成功的必要条件.尽管病毒载体的转化效率很高,他们的靶向性却往往无法满足治疗要求.过去这种靶向性的缺乏使得基因治疗的巨大潜力仅仅得到部分开发.最近通过各种技术对病毒载体改造的稳步进展使得这些载体在体外对许多细胞的靶性作用成为可能.虽然仍然存在诸多挑战,第一个利用靶向性载体的临床实验已经开始进行了.
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