Drug delivery trends in clinical trials and translational medicine: challenges and opportunities in the delivery of nucleic acid-based therapeutics.
Drug delivery trends in clinical trials and translational medicine: challenges and opportunities in the delivery of nucleic acid-based therapeutics.
复制标题
DOI：
10.1002/jps.22243
复制
发表时间：
2011-01
影响因子：
3.8
通讯作者：
Anchordoquy, Thomas
中科院分区：
文献类型：
--
作者：
Xu, Long;Anchordoquy, Thomas
关键词：
递送核酸（例如质粒 DNA、反义寡核苷酸、siRNA）的能力为开发有效的疫苗和新型疗法提供了潜力。然而，作为一种新的生物制剂，基于核酸的疗法仍处于早期阶段。核酸的功效需要将这些分子递送到靶细胞内部，这使递送策略变得非常复杂并降低了效率。由于病毒载体的安全性问题，脂质体和聚合物等合成载体优选用于递送基于核酸的治疗剂。然而，合成载体在临床上的递送效率仍然太低，无法获得治疗水平的基因表达。在这篇综述中，我们重点讨论了核酸递送领域的一些关键问题，例如聚乙二醇化、封装和靶向递送，并为开发改进的合成载体提供了一些考虑的观点。
The ability to deliver nucleic acids (e.g., plasmid DNA, antisense oligonucleotides, siRNA) offers the potential to develop potent vaccines and novel therapeutics. However, nucleic acid-based therapeutics are still in their early stages as a new category of biologics. The efficacy of nucleic acids requires that these molecules be delivered to the interior of the target cell, which greatly complicates delivery strategies and compromises efficiency. Due to the safety concerns of viral vectors, synthetic vectors such as liposomes and polymers are preferred for the delivery of nucleic acid-based therapeutics. Yet, delivery efficiencies of synthetic vectors in the clinic are still too low to obtain therapeutic levels of gene expression. In this review, we focus on some key issues in the field of nucleic acid delivery such as PEGylation, encapsulation and targeted delivery and provide some perspectives for consideration in the development of improved synthetic vectors.
登录
查看更多内容
影响因子：
3.4
作者：
Dos Santos, Nancy;Allen, Christine;Bally, Marcel B.
通讯作者：
Bally, Marcel B.
影响因子：
12.4
作者：
Akinc, Akin;Goldberg, Michael;Anderson, Daniel G.
通讯作者：
Anderson, Daniel G.
影响因子：
3.6
作者：
Eichhorn, M. E.;Luedemann, S.;Dellian, M.
通讯作者：
Dellian, M.
影响因子：
10.8
作者：
Deshpande, MC;Davies, MC;Stolnik, S
通讯作者：
Stolnik, S
影响因子：
56.9
作者：
Cartier, Nathalie;Hacein-Bey-Abina, Salima;Aubourg, Patrick
通讯作者：
Aubourg, Patrick