Editing cells in place: Targeted in vivo gene editing of hematopoietic stem cells

IF 23.3 1区医学 Q1 CELL & TISSUE ENGINEERING

Cell stem cell Pub Date : 2026-10-01 DOI:10.1016/j.stem.2026.08.015

Sijin Luozhong,Angelica Varesi,Bowen Li

引用次数: 0

Abstract

Traditional ex vivo hematopoietic stem cell (HSC) gene therapy faces manufacturing challenges. A recent study1 in Nature Biomedical Engineering demonstrates that antibody-engineered lipid nanoparticles achieve efficient and durable in vivo editing of HSCs in humanized mice, offering a promising therapeutic strategy for genetic blood diseases.

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就地编辑细胞：针对造血干细胞的体内基因编辑

传统的体外造血干细胞（HSC）基因治疗面临着制造上的挑战。《自然生物医学工程》杂志最近的一项研究表明，抗体工程脂质纳米颗粒在人源化小鼠体内实现了高效和持久的造血干细胞编辑，为遗传性血液疾病提供了一种有希望的治疗策略。

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.