The accessory makes the drug: ASO-delivered siRNA enters the human CNS for ALS

IF 23.3 1区医学 Q1 CELL & TISSUE ENGINEERING

Cell stem cell Pub Date : 2026-10-01 DOI:10.1016/j.stem.2026.09.004

Wenxuan Guo,Justin K. Ichida

引用次数: 0

Abstract

In a recent article in Nature Medicine, Chen et al. describe a first-in-human study using an ASO tag to deliver a SOD1 siRNA to the central nervous system (CNS), potentially achieving faster, more extensive, and better-tolerated SOD1-lowering in ALS patients.1

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药物的制造过程是：aso递送的siRNA进入人体中枢神经系统治疗ALS

在《自然医学》杂志最近的一篇文章中，Chen等人描述了一项首次使用ASO标签向中枢神经系统（CNS）递送SOD1 siRNA的人体研究，可能在ALS患者中实现更快、更广泛和更好耐受的SOD1降低

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Cell stem cell 生物-细胞生物学

CiteScore

37.10

自引率

2.50%

发文量

151

审稿时长

42 days

期刊介绍： Cell Stem Cell is a comprehensive journal covering the entire spectrum of stem cell biology. It encompasses various topics, including embryonic stem cells, pluripotency, germline stem cells, tissue-specific stem cells, differentiation, epigenetics, genomics, cancer stem cells, stem cell niches, disease models, nuclear transfer technology, bioengineering, drug discovery, in vivo imaging, therapeutic applications, regenerative medicine, clinical insights, research policies, ethical considerations, and technical innovations. The journal welcomes studies from any model system providing insights into stem cell biology, with a focus on human stem cells. It publishes research reports of significant importance, along with review and analysis articles covering diverse aspects of stem cell research.