Department of Chemistry, Tufts University, Medford, Massachusetts, 02155, USA.
Chembiochem. 2023 May 2;24(9):e202300009. doi: 10.1002/cbic.202300009. Epub 2023 Apr 4.
A major limitation for the development of more effective oligonucleotide therapeutics has been a lack of understanding of their penetration into the cytosol. While prior work has shown how backbone modifications affect cytosolic penetration, it is unclear how cytosolic penetration is affected by other features including base composition, base sequence, length, and degree of secondary structure. We have applied the chloroalkane penetration assay, which exclusively reports on material that reaches the cytosol, to investigate the effects of these characteristics on the cytosolic uptake of druglike oligonucleotides. We found that base composition and base sequence had moderate effects, while length did not correlate directly with the degree of cytosolic penetration. Investigating further, we found that the degree of secondary structure had the largest and most predictable correlations with cytosolic penetration. These methods and observations add a layer of design for maximizing the efficacy of new oligonucleotide therapeutics.
开发更有效的寡核苷酸治疗药物的主要限制因素是缺乏对其进入细胞质的理解。虽然之前的工作已经表明了骨架修饰如何影响细胞质渗透,但尚不清楚包括碱基组成、碱基序列、长度和二级结构程度在内的其他特征如何影响细胞质渗透。我们应用氯烷烃渗透测定法,该方法专门报告到达细胞质的物质,来研究这些特征对类似药物的寡核苷酸进入细胞质的影响。我们发现碱基组成和碱基序列有中等影响,而长度与细胞质渗透程度没有直接相关性。进一步研究发现,二级结构的程度与细胞质渗透的相关性最大且最可预测。这些方法和观察结果为最大限度地提高新寡核苷酸治疗药物的疗效增加了一层设计。
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