Excipients for nucleic acids delivery formulations - what happens after cell entry?

将核酸（NA）的使用从疫苗转移到其他疾病的治疗将需要细胞内递送水平的逐步改变。为了实现这一目标，赋形剂的作用是必不可少的。本文建议，除了NA细胞内生物利用度外，制剂中使用的赋形剂的细胞内行为和作用将通过监测不同的细胞过程和“标记物”来评估。最先进的分子生物学技术将用于获得这些过程的详细了解。

Moving the use of nucleic acid (NA) from vaccines to therapy of other diseases would require a step change in the level of intracellular delivery. To achieve that, the role of excipients is essential. It is proposed here that, in addition to NA intracellular bioavailability, the intracellular behaviour and effects of excipients used in formulation will be assessed by monitoring different cellular processes and 'markers'. State-of-the-art molecular biology techniques will be used to obtain a detailed understanding of these processes.