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癫痫治疗的新工具。

New Tools for Epilepsy Therapy.

作者信息

Falcicchia Chiara, Simonato Michele, Verlengia Gianluca

机构信息

Department of Medical Sciences, Section of Pharmacology, and Neuroscience Center, University of Ferrara and National Institute of Neuroscience, Ferrara, Italy.

School of Medicine, University Vita-Salute San Raffaele, Milan, Italy.

出版信息

Front Cell Neurosci. 2018 May 29;12:147. doi: 10.3389/fncel.2018.00147. eCollection 2018.

DOI:10.3389/fncel.2018.00147
PMID:29896092
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC5986878/
Abstract

One third of the epilepsies are refractory to conventional antiepileptic drugs (AEDs) and, therefore, identification of new therapies is highly needed. Here, we briefly describe two approaches, direct cell grafting and gene therapy, that may represent alternatives to conventional drugs for the treatment of focal epilepsies. In addition, we discuss more in detail some new tools, cell based-biodelivery systems (encapsulated cell biodelivery (ECB) devices) and new generation gene therapy vectors, which may help in the progress toward clinical translation. The field is advancing rapidly, and there is optimism that cell and/or gene therapy strategies will soon be ready for testing in drug-resistant epileptic patients.

摘要

三分之一的癫痫患者对传统抗癫痫药物(AEDs)耐药,因此,迫切需要找到新的治疗方法。在此,我们简要介绍两种可能替代传统药物治疗局灶性癫痫的方法,即直接细胞移植和基因治疗。此外,我们将更详细地讨论一些新工具,基于细胞的生物递送系统(封装细胞生物递送(ECB)装置)和新一代基因治疗载体,它们可能有助于推动临床转化进程。该领域发展迅速,人们乐观地认为细胞和/或基因治疗策略很快将准备好在耐药癫痫患者中进行测试。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/fab0/5986878/ff902bdc5e62/fncel-12-00147-g0002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/fab0/5986878/e80ab0556220/fncel-12-00147-g0001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/fab0/5986878/ff902bdc5e62/fncel-12-00147-g0002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/fab0/5986878/e80ab0556220/fncel-12-00147-g0001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/fab0/5986878/ff902bdc5e62/fncel-12-00147-g0002.jpg

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本文引用的文献

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Seizure-Suppressant and Neuroprotective Effects of Encapsulated BDNF-Producing Cells in a Rat Model of Temporal Lobe Epilepsy.封装的产生脑源性神经营养因子的细胞在颞叶癫痫大鼠模型中的抗癫痫和神经保护作用
Mol Ther Methods Clin Dev. 2018 Mar 9;9:211-224. doi: 10.1016/j.omtm.2018.03.001. eCollection 2018 Jun 15.
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Regulatory and Scientific Advancements in Gene Therapy: State-of-the-Art of Clinical Applications and of the Supporting European Regulatory Framework.基因治疗的监管与科学进展:临床应用及欧洲支持性监管框架的现状
Front Med (Lausanne). 2017 Oct 26;4:182. doi: 10.3389/fmed.2017.00182. eCollection 2017.
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Synthetic AAV/CRISPR vectors for blocking HIV-1 expression in persistently infected astrocytes.
用于阻断持续性感染星形胶质细胞中 HIV-1 表达的合成 AAV/CRISPR 载体。
Glia. 2018 Feb;66(2):413-427. doi: 10.1002/glia.23254. Epub 2017 Nov 9.
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Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy.脊髓性肌萎缩症的单剂量基因治疗。
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Persistent seizure control in epileptic mice transplanted with gamma-aminobutyric acid progenitors.移植γ-氨基丁酸祖细胞的癫痫小鼠的持续性癫痫控制
Ann Neurol. 2017 Oct;82(4):530-542. doi: 10.1002/ana.25021. Epub 2017 Oct 4.
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Deletion of the Virion Host Shut-off Gene Enhances Neuronal-Selective Transgene Expression from an HSV Vector Lacking Functional IE Genes.病毒体宿主关闭基因的缺失增强了来自缺乏功能性立即早期基因的单纯疱疹病毒载体的神经元选择性转基因表达。
Mol Ther Methods Clin Dev. 2017 Jun 16;6:79-90. doi: 10.1016/j.omtm.2017.06.001. eCollection 2017 Sep 15.
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Engineered HSV vector achieves safe long-term transgene expression in the central nervous system.工程化 HSV 载体可在中枢神经系统中实现安全的长期转基因表达。
Sci Rep. 2017 May 4;7(1):1507. doi: 10.1038/s41598-017-01635-1.
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eNeuro. 2017 Apr 12;4(2). doi: 10.1523/ENEURO.0359-16.2017. eCollection 2017 Mar-Apr.
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Genes (Basel). 2017 Feb 10;8(2):65. doi: 10.3390/genes8020065.
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PLoS One. 2016 Mar 8;11(3):e0150995. doi: 10.1371/journal.pone.0150995. eCollection 2016.