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神经退行性疾病的基因治疗：放慢生命时钟

Gene Therapy for Neurodegenerative Diseases: Slowing Down the Ticking Clock.

作者信息

Martier Raygene, Konstantinova Pavlina

机构信息

Department of Research and Development, uniQure Biopharma B.V., Amsterdam, Netherlands.

出版信息

Front Neurosci. 2020 Sep 18;14:580179. doi: 10.3389/fnins.2020.580179. eCollection 2020.

DOI:10.3389/fnins.2020.580179
PMID:33071748
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC7530328/
Abstract

Gene therapy is an emerging and powerful therapeutic tool to deliver functional genetic material to cells in order to correct a defective gene. During the past decades, several studies have demonstrated the potential of AAV-based gene therapies for the treatment of neurodegenerative diseases. While some clinical studies have failed to demonstrate therapeutic efficacy, the use of AAV as a delivery tool has demonstrated to be safe. Here, we discuss the past, current and future perspectives of gene therapies for neurodegenerative diseases. We also discuss the current advances on the newly emerging RNAi-based gene therapies which has been widely studied in preclinical model and recently also made it to the clinic.

摘要

基因治疗是一种新兴且强大的治疗工具，可将功能性遗传物质传递到细胞中，以纠正有缺陷的基因。在过去几十年中，多项研究已证明基于腺相关病毒（AAV）的基因疗法在治疗神经退行性疾病方面的潜力。虽然一些临床研究未能证明其治疗效果，但将AAV用作递送工具已证明是安全的。在此，我们讨论神经退行性疾病基因治疗的过去、现状和未来前景。我们还讨论了新兴的基于RNA干扰（RNAi）的基因疗法的当前进展，该疗法已在临床前模型中得到广泛研究，最近也已进入临床应用。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/07ec/7530328/f111f36d0e02/fnins-14-580179-g005.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/07ec/7530328/c9bf96907eb6/fnins-14-580179-g001.jpg
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https://cdn.ncbi.nlm.nih.gov/pmc/blobs/07ec/7530328/5718ebc95c89/fnins-14-580179-g004.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/07ec/7530328/f111f36d0e02/fnins-14-580179-g005.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/07ec/7530328/c9bf96907eb6/fnins-14-580179-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/07ec/7530328/bc413d8bd50f/fnins-14-580179-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/07ec/7530328/0f1a954c2ef0/fnins-14-580179-g003.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/07ec/7530328/5718ebc95c89/fnins-14-580179-g004.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/07ec/7530328/f111f36d0e02/fnins-14-580179-g005.jpg

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