多 Q 疾病的分子疗法：从实验室到临床试验。

IF 12.8 1区医学 Q1 BIOCHEMISTRY & MOLECULAR BIOLOGY

Trends in molecular medicine Pub Date : 2024-09-01 Epub Date: 2024-06-04 DOI:10.1016/j.molmed.2024.05.004

João de Sousa-Lourenço, Ana C Silva, Luís Pereira de Almeida, Rui J Nobre

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引用次数: 0

摘要

多谷氨酰胺（polyQ）疾病是一种单基因神经退行性疾病。目前，还没有针对这类复杂疾病的疗法。在此，我们旨在概述近期前景看好的临床前研究和正在进行的临床试验，重点关注多聚 Q 紊乱症的分子疗法。

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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Molecular therapy for polyQ disorders: from bench to clinical trials.

Polyglutamine (polyQ) disorders are monogenic neurodegenerative disorders. Currently, no therapies are available for this complex group of disorders. Here, we aim to provide an overview of recent promising preclinical studies and the ongoing clinical trials focusing on molecular therapies for polyQ disorders.

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来源期刊

Trends in molecular medicine 医学-生化与分子生物学

CiteScore

24.60

自引率

0.00%

发文量

142

审稿时长

6-12 weeks

期刊介绍： Trends in Molecular Medicine (TMM) aims to offer concise and contextualized perspectives on the latest research advancing biomedical science toward better diagnosis, treatment, and prevention of human diseases. It focuses on research at the intersection of basic biology and clinical research, covering new concepts in human biology and pathology with clear implications for diagnostics and therapy. TMM reviews bridge the gap between bench and bedside, discussing research from preclinical studies to patient-enrolled trials. The major themes include disease mechanisms, tools and technologies, diagnostics, and therapeutics, with a preference for articles relevant to multiple themes. TMM serves as a platform for discussion, pushing traditional boundaries and fostering collaboration between scientists and clinicians. The journal seeks to publish provocative and authoritative articles that are also accessible to a broad audience, inspiring new directions in molecular medicine to enhance human health.