期刊
AAPS JOURNAL
卷 23, 期 4, 页码 -出版社
SPRINGER
DOI: 10.1208/s12248-021-00608-7
关键词
formulation; gene therapy; lipid; non-viral vector; polymer; transfection
Although non-viral vectors in gene therapy have low cytotoxicity and immunogenicity, they still face challenges in gene transfer efficiency, specificity, gene expression duration, and safety. Overcoming these obstacles is necessary to enhance the effectiveness of non-viral vectors in gene therapy.
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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