以N-of-1碱基编辑器治疗罕见疾病的势头为基础

IF 101.8 1区医学 Q1 BIOTECHNOLOGY & APPLIED MICROBIOLOGY

Nature Reviews. Drug Discovery Pub Date : 2025-07-15 DOI:10.1038/d41573-025-00119-6

Katie Kingwell

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引用次数: 0

摘要

费城儿童医院的医生兼科学家丽贝卡·阿伦斯-尼克拉斯（Rebecca Ahrens-Nicklas）讨论了她对一种超罕见尿素循环障碍的基因编辑疗法的突破性应用。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

Building on the momentum of N-of-1 base editor therapies for rare diseases

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Building on the momentum of N-of-1 base editor therapies for rare diseases

Rebecca Ahrens-Nicklas, a physician-scientist at the Children’s Hospital of Philadelphia, discusses her groundbreaking application of a gene-editing therapy for an ultra-rare urea cycle disorder. Rebecca Ahrens-Nicklas, a physician-scientist at the Children’s Hospital of Philadelphia, discusses her groundbreaking application of a gene-editing therapy for an ultra-rare urea cycle disorder.

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来源期刊

Nature Reviews. Drug Discovery 医学-生物工程与应用微生物

CiteScore

137.40

自引率

0.30%

发文量

227

期刊介绍： Nature Reviews Drug Discovery is a monthly journal aimed at everyone working in the drug discovery and development arena. Each issue includes: Highest-quality reviews and perspectives covering a broad scope. News stories investigating the hottest topics in drug discovery. Timely summaries of key primary research papers. Concise updates on the latest advances in areas such as new drug approvals, patent law, and emerging industry trends and strategies.