Engineering of a bespoke CRISPR base editor to treat a severe vascular disease

IF 26.8 1区医学 Q1 ENGINEERING, BIOMEDICAL

Nature Biomedical Engineering Pub Date : 2025-09-15 DOI:10.1038/s41551-025-01510-9

引用次数: 0

Abstract

We developed a customized base editing strategy to efficiently, precisely and safely correct the most common ACTA2 pathogenic mutation in multisystemic smooth muscle dysfunction syndrome. In vivo delivery of the bespoke base editor prolongs survival and rescues systemic phenotypes in a mouse model of multisystemic smooth muscle dysfunction syndrome.

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一种定制的CRISPR碱基编辑器用于治疗一种严重的血管疾病

我们开发了一种定制的碱基编辑策略，以有效、精确和安全地纠正多系统平滑肌功能障碍综合征中最常见的ACTA2致病突变。在多系统平滑肌功能障碍综合征小鼠模型中，体内递送定制的碱基编辑器延长了生存期并挽救了系统表型。

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

Nature Biomedical Engineering Medicine-Medicine (miscellaneous)

CiteScore

45.30

自引率

1.10%

发文量

138

期刊介绍： Nature Biomedical Engineering is an online-only monthly journal that was launched in January 2017. It aims to publish original research, reviews, and commentary focusing on applied biomedicine and health technology. The journal targets a diverse audience, including life scientists who are involved in developing experimental or computational systems and methods to enhance our understanding of human physiology. It also covers biomedical researchers and engineers who are engaged in designing or optimizing therapies, assays, devices, or procedures for diagnosing or treating diseases. Additionally, clinicians, who make use of research outputs to evaluate patient health or administer therapy in various clinical settings and healthcare contexts, are also part of the target audience.