Gene-based therapies for neuromuscular disorders.

IF 1 4区医学 Q4 NEUROSCIENCES

Arquivos de neuro-psiquiatria Pub Date : 2024-06-01 Epub Date: 2024-02-07 DOI:10.1055/s-0043-1777755

Edmar Zanoteli, Marcondes Cavalcante França, Wilson Marques

引用次数: 0

Abstract

Neuromuscular diseases (NMD) include a broad group of medical conditions with both acquired and genetic causes. In recent years, important advances have been made in the treatment of genetically caused NMD, and most of these advances are due to the implementation of therapies aimed at gene regulation. Among these therapies, gene replacement, small interfering RNA (siRNA), and antisense antinucleotides are the most promising approaches. More importantly, some of these therapies have already gained regulatory approval or are in the final stages of approval. The review focuses on motor neuron diseases, neuropathies, and Duchenne muscular dystrophy, summarizing the most recent developments in gene-based therapies for these conditions.

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基于基因的神经肌肉疾病疗法。

神经肌肉疾病（NMD）包括一大类既有获得性原因也有遗传性原因的病症。近年来，在治疗遗传性 NMD 方面取得了重大进展，而这些进展大多归功于旨在调节基因的疗法的实施。在这些疗法中，基因替换、小干扰 RNA（siRNA）和反义反核苷酸是最有前途的方法。更重要的是，其中一些疗法已经获得了监管部门的批准，或正处于审批的最后阶段。本综述以运动神经元疾病、神经病和杜氏肌营养不良症为重点，总结了针对这些疾病的基因疗法的最新进展。

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

Arquivos de neuro-psiquiatria 医学-精神病学

CiteScore

2.10

自引率

7.10%

发文量

262

审稿时长

4-8 weeks

期刊介绍： Arquivos de Neuro-Psiquiatria is the official journal of the Brazilian Academy of Neurology. The mission of the journal is to provide neurologists, specialists and researchers in Neurology and related fields with open access to original articles (clinical and translational research), editorials, reviews, historical papers, neuroimages and letters about published manuscripts. It also publishes the consensus and guidelines on Neurology, as well as educational and scientific material from the different scientific departments of the Brazilian Academy of Neurology. The ultimate goals of the journal are to contribute to advance knowledge in the areas of Neurology and Neuroscience, and to provide valuable material for training and continuing education for neurologists and other health professionals working in the area. These goals might contribute to improving care for patients with neurological diseases. We aim to be the best Neuroscience journal in Latin America within the peer review system.