由阳离子b[4]螺旋烯-角鲨烯酯自组装的两亲性纳米载体在线粒体靶向递送siRNA。

IF 2.8 4区生物学 Q3 BIOCHEMISTRY & MOLECULAR BIOLOGY

ChemBioChem Pub Date : 2025-10-07 DOI:10.1002/cbic.202500537

Lamiaa M A Ali, Dan-Dan Su, Rebecca Mucci, Kévin Martin, Mihail Barboiu, Jérôme Lacour, Nadir Bettache, Sébastien Ulrich

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引用次数: 0

摘要

具有亚细胞精度的治疗性核酸的主动递送是一个公开的挑战。在这里，一个小的共轭分子，结合了一个阳离子b[4]螺旋烯片段和亲脂性角鲨烯尾部，产生了两亲性纳米组件，能够在线粒体内有效地络合siRNA。Western blot证实，在HCT 116结肠直肠癌细胞中，siRNA的传递可以沉默线粒体编码的细胞色素c氧化酶亚基1基因的蛋白表达。

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Mitochondria-Targeted Delivery of siRNA by Amphiphilic Nanovectors Self-Assembled from a Cationic [4]Helicene-Squalene Ester.

The active delivery of therapeutic nucleic acids with subcellular precision is an open challenge. Herein, a small conjugate molecule, combining a cationic [4]helicene moiety with a lipophilic squalene tail, yields amphiphilic nanoassemblies capable of effectively complexing siRNA within the mitochondria. A proof-of-concept in HCT 116 colorectal cancer cells was evidenced by Western blot for the delivery of siRNA silencing the protein expression of the mitochondria-encoded cytochrome c oxidase subunit 1 gene.

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来源期刊

ChemBioChem 生物-生化与分子生物学

CiteScore

6.10

自引率

3.10%

发文量

407

审稿时长

1 months

期刊介绍： ChemBioChem (Impact Factor 2018: 2.641) publishes important breakthroughs across all areas at the interface of chemistry and biology, including the fields of chemical biology, bioorganic chemistry, bioinorganic chemistry, synthetic biology, biocatalysis, bionanotechnology, and biomaterials. It is published on behalf of Chemistry Europe, an association of 16 European chemical societies, and supported by the Asian Chemical Editorial Society (ACES).