Polymer-mediated gene therapy: Recent advances and merging of delivery techniques.

Polymer-mediated gene therapy: Recent advances and merging of delivery techniques.
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DOI:
10.1002/wnan.1598
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发表时间:
2020-03
期刊:
Wiley interdisciplinary reviews. Nanomedicine and nanobiotechnology
影响因子:
--
通讯作者:
Figueiredo ML
Figueiredo ML
中科院分区:
其他
文献类型:
--
作者:
Salameh JW;Zhou L;Ward SM;Santa Chalarca CF;Emrick T;Figueiredo ML

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在复杂的生物环境中,将遗传物质安全、精确地递送到靶位点的能力对于基因治疗的成功至关重要。已经开发了许多病毒和非病毒载体,并对其安全性和有效性进行了评价。本研究将介绍合成聚合物作为非病毒载体的进展,这些聚合物受益于其化学多功能性,生物相容性以及携带治疗货物和靶向部分的能力。携带合成基因的构建体与先进的递送技术的组合为治疗和治愈遗传疾病提供了新的治疗选择。
The ability to safely and precisely deliver genetic materials to target sites in complex biological environments is vital to the success of gene therapy. Numerous viral and nonviral vectors have been developed and evaluated for their safety and efficacy. This study will feature progress in synthetic polymers as nonviral vectors, which benefit from their chemical versatility, biocompatibility, and ability to carry both therapeutic cargo and targeting moieties. The combination of synthetic gene carrying constructs with advanced delivery techniques promises new therapeutic options for treating and curing genetic disorders.
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