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肌萎缩侧索硬化症的基因治疗

Gene therapy of amyotrophic lateral sclerosis.

作者信息

Zavalishin I A, Bochkov N P, Suslina Z A, Zakharova M N, Tarantul V Z, Naroditskiy B S, Suponeva N A, Illarioshkin S N, Shmarov M M, Logunov D Y, Tutyhina I L, Verkhovskaya L V, Sedova E S, Vasiliev A V, Brylev L V, Ginzburg A L

机构信息

Research Center of Neurology, Russian Academy of Medical Sciences, Russia.

出版信息

Bull Exp Biol Med. 2008 Apr;145(4):483-6. doi: 10.1007/s10517-008-0124-4.

DOI:10.1007/s10517-008-0124-4
PMID:19110600
Abstract

Two-year experiments were performed to evaluate the neurotrophic effect of hypoxia-inducible factors (vascular endothelial growth factor and angiogenin) expressed in recombinant human adenoviruses in amyotrophic lateral sclerosis. Randomized placebo-controlled trial demonstrated safety and good tolerability of the recombinant antiviral drugs. The life span of patients under conditions of hypoxia increased after treatment with the test drug, which was probably related to improved resistance of motoneurons. The presence of virus-neutralizing antibodies decreases the effectiveness of adenoviral vectors, which necessitates differential approach to the selection of patients and continuous monitoring of gene therapy.

摘要

进行了为期两年的实验,以评估重组人腺病毒中表达的缺氧诱导因子(血管内皮生长因子和血管生成素)在肌萎缩侧索硬化症中的神经营养作用。随机安慰剂对照试验证明了重组抗病毒药物的安全性和良好耐受性。用受试药物治疗后,缺氧条件下患者的寿命延长,这可能与运动神经元抵抗力的提高有关。病毒中和抗体的存在会降低腺病毒载体的有效性,这就需要在患者选择上采用差异化方法并持续监测基因治疗。

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引用本文的文献

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Neural Regen Res. 2021 Mar;16(3):550-560. doi: 10.4103/1673-5374.293150.
2
Spinal Cord Molecular and Cellular Changes Induced by Adenoviral Vector- and Cell-Mediated Triple Gene Therapy after Severe Contusion.严重挫伤后腺病毒载体和细胞介导的三联基因治疗诱导的脊髓分子和细胞变化
Front Pharmacol. 2017 Nov 13;8:813. doi: 10.3389/fphar.2017.00813. eCollection 2017.
3
Research advances in gene therapy approaches for the treatment of amyotrophic lateral sclerosis.
肌萎缩侧索硬化症基因治疗方法的研究进展。
Cell Mol Life Sci. 2012 May;69(10):1641-50. doi: 10.1007/s00018-011-0881-5. Epub 2011 Nov 18.
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A neuroprotective role for angiogenin in models of Parkinson's disease.血管生成素在帕金森病模型中的神经保护作用。
J Neurochem. 2011 Feb;116(3):334-41. doi: 10.1111/j.1471-4159.2010.07112.x. Epub 2010 Dec 22.