二比一好：推进基因治疗方案，提高安全性和有效性

IF 19.8 1区医学 Q1 CELL & TISSUE ENGINEERING

Cell stem cell Pub Date : 2025-02-06 DOI:10.1016/j.stem.2024.12.009

Giulia Hardouin, Annarita Miccio

引用次数: 0

摘要

在本期《Cell Stem Cell》中，Zeng et al.1和Demirci et al.2的两项互补研究利用多重CRISPR-Cas9编辑技术提高了基因治疗β-血红蛋白病的疗效。作者还优化了编辑和调节方案，以降低与当前基因治疗程序相关的风险。

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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Two is better than one: Advancing gene therapy protocols for enhanced safety and efficacy

In this issue of Cell Stem Cell, two complementary studies from Zeng et al.¹ and Demirci et al.² improve the efficacy of gene therapy for β-hemoglobinopathies using multiplex CRISPR-Cas9 editing. The authors also optimize editing and conditioning protocols to reduce risks associated with current gene therapy procedures.

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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.