Gene therapy of amyotrophic lateral sclerosis
Gene therapy of amyotrophic lateral sclerosis
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肌萎缩侧索硬化症的基因治疗
DOI：
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发表时间：
2008
影响因子：
0.7
通讯作者：
A. Ginzburg
中科院分区：
文献类型：
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作者：
I. Zavalishin;N. Bochkov;Z. Suslina;M. Zakharova;V. Tarantul;B. S. Naroditskiy;N. Suponeva;S. Illarioshkin;M. Shmarov;D. Logunov;I. L. Tutyhina;L. V. Verkhovskaya;E. Sedova;A. Vasiliev;L. Brylev;A. Ginzburg
进行了为期两年的实验，以评估缺氧诱导因子（血管内皮生长因子和血管生成素）表达的重组人腺病毒在肌萎缩侧索硬化症的神经营养作用。随机安慰剂对照试验证实了重组抗病毒药物的安全性和良好的耐受性。试验药物治疗后，患者在缺氧条件下的寿命延长，这可能与运动神经元的抵抗力改善有关。病毒中和抗体的存在降低了腺病毒载体的有效性，这就需要对患者的选择和基因治疗的持续监测采取不同的方法。
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.