小鼠鞘内U1接头寡核苷酸的组织分布及药代动力学

Q2 Agricultural and Biological Sciences

Current Research in Pharmacology and Drug Discovery Pub Date : 2025-01-01 DOI:10.1016/j.crphar.2025.100220

Medha Satti , Kavita Prasad , Yash Patel , Demi Poulathas , Lawrence Walker , Esha Paghdal , Samuel Gunderson , Lei Yu

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引用次数: 0

摘要

U1 Adaptor是一种新型的基因沉默技术，为中枢神经系统中的靶基因治疗疾病提供了一种创新的方法。鞘内给药是医学上可行的给药途径；因此，研究鞘内给药后U1 Adaptor的分布是很重要的。我们研究了小鼠鞘内给药后U1 Adaptor的分布模式。它很容易分布到中枢神经系统组织，包括腰、颈脊髓和小脑。随着时间的推移，U1适配器也会在肝脏和肾脏的外周积聚，而血浆水平则无法检测到。我们的发现为未来深入建立鞘内给药U1 Adaptor分布的药代动力学模型提供了有用的信息。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

Tissue distribution pharmacokinetics of intrathecal U1 adaptor oligonucleotide in mice

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Tissue distribution pharmacokinetics of intrathecal U1 adaptor oligonucleotide in mice

U1 Adaptor is a novel gene-silencing technology, offering an innovative approach to target genes in the CNS for the treatment of diseases. Intrathecal delivery is a medically viable route of administration of CNS-bound nucleic acid drugs; therefore, it is important to investigate U1 Adaptor distribution after intrathecal drug delivery. We investigated the distribution patterns of U1 Adaptor upon intrathecal bolus administration in mice. It readily distributes to CNS tissues, including the lumbar and the cervical spinal cord, and the cerebellum. Over time, the U1 Adaptor also accumulates in the periphery, both in the liver and the kidneys, while plasma levels are undetectable. Our findings provide useful information for future in-depth pharmacokinetic modeling of U1 Adaptor distribution upon intrathecal administration.

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来源期刊

Current Research in Pharmacology and Drug Discovery Agricultural and Biological Sciences-Animal Science and Zoology

CiteScore

6.40

自引率

0.00%

发文量

审稿时长

40 days