Amgen Research, Pharmacokinetics & Drug Metabolism, Amgen Inc., South San Francisco, CA, US.
Sci Rep. 2020 Jun 26;10(1):10425. doi: 10.1038/s41598-020-66829-6.
Oligonucleotide therapeutics use short interfering RNA (siRNA) or antisense oligonucleotide (ASO) molecules to exploit endogenous systems-neutralizing target RNA to prevent subsequent protein translation. While the potential clinical application is vast, delivery efficiency and extrahepatic targeting is challenging. Bioanalytical assays are important in building understanding of these complex relationships. The literature currently lacks description of robust and sensitive methods to measure siRNA and ASOs in complex biological matrices. Described herein is a non-enzymatic hybridization-based immunoassay that enables quantification of individual siRNA strands (antisense or sense) in serum, urine, bile, and liver and kidney homogenates. Assay utility is also demonstrated in ASOs. The assay improves upon previous works by abolishing enzymatic steps and further incorporating Locked Nucleic Acid (LNA) nucleotide modifications to increase analyte hybridization affinity and improve sensitivity, specificity, and robustness. We report an assay with an ultrasensitive dynamic range of 0.3 to 16,700 pM for siRNA in serum. The assay was submitted to full qualification for accuracy and precision in both serum and tissue matrices and assay performance was assessed with single and mixed analytes. The reliable LNA-hybridization-based approach removes the need for matrix sample extraction, enrichment or amplification steps which may be impeded by more advanced chemical modifications.
寡核苷酸疗法利用短干扰 RNA (siRNA) 或反义寡核苷酸 (ASO) 分子来利用内源性系统中和靶 RNA,以防止随后的蛋白质翻译。虽然潜在的临床应用是广泛的,但输送效率和肝外靶向是具有挑战性的。生物分析测定在深入了解这些复杂关系方面非常重要。目前,文献中缺乏描述用于测量复杂生物基质中 siRNA 和 ASO 的稳健和敏感方法的描述。本文描述了一种基于非酶杂交的免疫测定法,可用于定量血清、尿液、胆汁和肝、肾匀浆中的单个 siRNA 链(反义或正义)。该测定法在 ASO 中的应用也得到了证明。该测定法通过消除酶促步骤并进一步引入锁定核酸 (LNA) 核苷酸修饰来提高分析物杂交亲和力,从而提高灵敏度、特异性和稳健性,改进了以前的工作。我们报告了一种在血清中具有超灵敏动态范围为 0.3 至 16700 pM 的 siRNA 测定法。该测定法已提交用于在血清和组织基质中进行准确性和精密度的全面资格认证,并且使用单一和混合分析物评估了测定法的性能。可靠的基于 LNA 杂交的方法消除了对基质样品提取、富集或扩增步骤的需求,这些步骤可能会受到更先进的化学修饰的阻碍。