Adeno-associated virus serotypes for gene therapeutics
Adeno-associated virus serotypes for gene therapeutics
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DOI：
10.1016/j.coph.2015.07.006
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发表时间：
2015-10-01
影响因子：
4
通讯作者：
Alexander, Ian Edward
中科院分区：
文献类型：
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作者：
Lisowski, Leszek;Tay, Szun Szun;Alexander, Ian Edward
基于腺相关病毒 (AAV) 的基因转移载体在早期临床试验中显示出令人兴奋的治疗前景。将原型 AAV2 载体基因组交叉包装到不同衣壳中的能力是赋予新趋向性和生物学的强大方法，不断发展的衣壳工程技术和定向进化方法进一步增强了这些载体的实用性和灵活性。特定衣壳的新特性显示出不可预测的物种和细胞类型特异性。因此，充分实现 AAV 载体的治疗潜力需要开发更具治疗预测性的临床前方法来评估衣壳性能。这将有力地补充临床中衣壳变异评估的迭代方法，并且应尽可能包括基因转移效率的测定。
Gene transfer vectors based on adeno-associated virus (AAV) are showing exciting therapeutic promise in early phase clinical trials. The ability to cross-package the prototypic AAV2 vector genome into different capsids is a powerful way of conferring novel tropism and biology, with evolving capsid engineering technologies and directed evolution approaches further enhancing the utility and flexibility of these vectors. Novel properties of specific capsids show unpredictable species and cell-type specificity. Therefore, full realisation of the therapeutic potential of AAV vectors requires the development of more therapeutically predictive preclinical methods for evaluating capsid performance. This will strongly complement an iterative approach to the evaluation of capsid variants in the clinic and, should wherever possible, include the determination of gene transfer efficiencies.