静脉注射可跨越血脑屏障的共轭物，促进生物大分子向中枢神经系统的转运

IF 33.1 1区生物学 Q1 BIOTECHNOLOGY & APPLIED MICROBIOLOGY

Nature biotechnology Pub Date : 2024-11-25 DOI:10.1038/s41587-024-02487-7

Chang Wang, Siyu Wang, Yonger Xue, Yichen Zhong, Haoyuan Li, Xucheng Hou, Diana D. Kang, Zhengwei Liu, Meng Tian, Leiming Wang, Dinglingge Cao, Yang Yu, Jayce Liu, Xiaolin Cheng, Tamara Markovic, Alice Hashemi, Brian H. Kopell, Alexander W. Charney, Eric J. Nestler, Yizhou Dong

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引用次数: 0

摘要

由于血脑屏障（BBB）的限制性，向中枢神经系统（CNS）输送生物大分子仍然具有挑战性。我们开发了一种跨血脑屏障共轭物（BCC）系统，可通过γ-分泌酶介导的转囊作用将生物大分子送入中枢神经系统。静脉注射 BCC10-寡核苷酸共轭物后，野生型小鼠、人类脑组织和肌萎缩性侧索硬化症小鼠模型中的寡核苷酸均能有效穿越 BBB，并实现基因沉默。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

Intravenous administration of blood–brain barrier-crossing conjugates facilitate biomacromolecule transport into central nervous system

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Intravenous administration of blood–brain barrier-crossing conjugates facilitate biomacromolecule transport into central nervous system

Delivery of biomacromolecules to the central nervous system (CNS) remains challenging because of the restrictive nature of the blood–brain barrier (BBB). We developed a BBB-crossing conjugate (BCC) system that facilitates delivery into the CNS through γ-secretase-mediated transcytosis. Intravenous administration of a BCC10–oligonucleotide conjugate demonstrated effective transportation of the oligonucleotide across the BBB and gene silencing in wild-type mice, human brain tissues and an amyotrophic lateral sclerosis mouse model.

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来源期刊

Nature biotechnology 工程技术-生物工程与应用微生物

CiteScore

63.00

自引率

1.70%

发文量

382

审稿时长

3 months

期刊介绍： Nature Biotechnology is a monthly journal that focuses on the science and business of biotechnology. It covers a wide range of topics including technology/methodology advancements in the biological, biomedical, agricultural, and environmental sciences. The journal also explores the commercial, political, ethical, legal, and societal aspects of this research. The journal serves researchers by providing peer-reviewed research papers in the field of biotechnology. It also serves the business community by delivering news about research developments. This approach ensures that both the scientific and business communities are well-informed and able to stay up-to-date on the latest advancements and opportunities in the field. Some key areas of interest in which the journal actively seeks research papers include molecular engineering of nucleic acids and proteins, molecular therapy, large-scale biology, computational biology, regenerative medicine, imaging technology, analytical biotechnology, applied immunology, food and agricultural biotechnology, and environmental biotechnology. In summary, Nature Biotechnology is a comprehensive journal that covers both the scientific and business aspects of biotechnology. It strives to provide researchers with valuable research papers and news while also delivering important scientific advancements to the business community.