免疫原性
病毒载体
遗传增强
基因传递
计算生物学
基因治疗载体
生物
基因转移
载体(分子生物学)
基因
免疫学
遗传学
免疫系统
重组DNA
出处
期刊:Aaps Journal
[Springer Nature]
日期:2021-06-02
卷期号:23 (4)
被引量:167
标识
DOI:10.1208/s12248-021-00608-7
摘要
Gene therapy has been experiencing a breakthrough in recent years, targeting various specific cell groups in numerous therapeutic areas. However, most recent clinical studies maintain the use of traditional viral vector systems, which are challenging to manufacture cost-effectively at a commercial scale. Non-viral vectors have been a fast-paced research topic in gene delivery, such as polymers, lipids, inorganic particles, and combinations of different types. Although non-viral vectors are low in their cytotoxicity, immunogenicity, and mutagenesis, attracting more and more researchers to explore the promising delivery system, they do not carry ideal characteristics and have faced critical challenges, including gene transfer efficiency, specificity, gene expression duration, and safety. This review covers the recent advancement in non-viral vectors research and formulation aspects, the challenges, and future perspectives.
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