In Vivo Selection Against Human Colorectal Cancer Xenografts Identifies an Aptamer That Targets RNA Helicase Protein DHX9

针对人类结直肠癌异种移植的体内筛选鉴定出靶向 RNA 解旋酶蛋白 DHX9 的适体

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作者:Jing Mi, Partha Ray, Jenny Liu, Chien-Tsun Kuan, Jennifer Xu, David Hsu, Bruce A Sullenger, Rebekah R White, Bryan M Clary

Abstract

The ability to selectively target disease-related tissues with molecules is critical to the design of effective therapeutic and diagnostic reagents. Recognizing the differences between the in vivo environment and in vitro conditions, we employed an in vivo selection strategy to identify RNA aptamers (targeting motifs) that could localize to tumor in situ. One of the selected molecules is an aptamer that binds to the protein DHX9, an RNA helicase that is known to be upregulated in colorectal cancer. Upon systemic administration, the aptamer preferentially localized to the nucleus of cancer cells in vivo and thus has the potential to be used for targeted delivery.

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