Silencing the mutant allele without compromising gene dosage

IF 23.3 1区医学 Q1 CELL & TISSUE ENGINEERING

Cell stem cell Pub Date : 2026-10-01 DOI:10.1016/j.stem.2026.08.011

Lu Tian,Junhan Chen,Yinqing Li

引用次数: 0

Abstract

Allele-selective silencing offers a strategy to suppress pathogenic alleles in dominant genetic disorders but may alter total gene dosage when the remaining allele does not compensate. Wang et al.1 show that SNP-guided epigenetic repression of mutant alleles can elicit compensatory expression from non-targeted alleles, highlighting considerations for future therapeutic applications.

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沉默突变等位基因而不影响基因剂量

等位基因选择性沉默提供了一种抑制显性遗传疾病致病性等位基因的策略，但当剩余等位基因无法补偿时，可能会改变基因的总剂量。Wang等人1表明，snp引导的突变等位基因的表观遗传抑制可以引起非靶向等位基因的代偿性表达，这突出了对未来治疗应用的考虑。

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.