耐药局灶性癫痫基因治疗的最新进展和挑战。

IF 2.8 4区医学 Q2 CLINICAL NEUROLOGY

Revue neurologique Pub Date : 2025-03-29 DOI:10.1016/j.neurol.2025.02.008

D M Kullmann

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引用次数: 0

摘要

耐药局灶性癫痫是一个未得到满足的主要需求。近年来，几种基因治疗策略主要在颞叶癫痫的啮齿动物模型中得到验证，其中一些已被用于临床试验。这篇综述考虑了从实验模型到临床进展中的一些挑战。其中包括确定有希望的启动子-转基因组合，建立安全有效的剂量，实现最佳递送，以及推断不同的病因。

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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Recent progress and challenges in gene therapy for pharmacoresistant focal epilepsy.

Pharmacoresistant focal epilepsy represents a major unmet need. Recent years have seen several gene therapy strategies validated mainly in rodent models of temporal lobe epilepsy, and some of these have been de-risked for clinical trials. This review considers some of the challenges in progressing from experimental models to the clinic. Among these are identifying promising promoter-transgene combinations, establishing safe and efficacious doses, achieving optimal delivery, and extrapolating across different aetiologies.

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来源期刊

Revue neurologique 医学-临床神经学

CiteScore

4.80

自引率

0.00%

发文量

598

审稿时长

55 days

期刊介绍： The first issue of the Revue Neurologique, featuring an original article by Jean-Martin Charcot, was published on February 28th, 1893. Six years later, the French Society of Neurology (SFN) adopted this journal as its official publication in the year of its foundation, 1899. The Revue Neurologique was published throughout the 20th century without interruption and is indexed in all international databases (including Current Contents, Pubmed, Scopus). Ten annual issues provide original peer-reviewed clinical and research articles, and review articles giving up-to-date insights in all areas of neurology. The Revue Neurologique also publishes guidelines and recommendations. The Revue Neurologique publishes original articles, brief reports, general reviews, editorials, and letters to the editor as well as correspondence concerning articles previously published in the journal in the correspondence column.