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首页> 外文期刊>Expert opinion on therapeutic targets >Targeting angiogenin in therapy of amyotropic lateral sclerosis.
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Targeting angiogenin in therapy of amyotropic lateral sclerosis.

机译：在肌萎缩性侧索硬化症的治疗中靶向血管生成。

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摘要

BACKGROUND: Missense heterozygous mutations in the coding region of angiogenin (ANG) gene, encoding a 14 kDa angiogenic RNase, were recently found in patients of amyotropic lateral sclerosis (ALS). Functional analyses have shown that these are loss-of-function mutations, implying that angiogenin deficiency is associated with ALS pathogenesis and that increasing ANG expression or angiogenin activity could be a novel approach for ALS therapy. OBJECTIVE: Review the evidence showing the involvement of angiogenin in motor neuron physiology and function, and provide a rationale for targeting angiogenin in ALS therapy. METHODS: Review the current understanding of the mechanism of angiogenin action in connection with ALS genetics, pathogenesis and therapy. CONCLUSION: ANG is the first gene whose loss-of-function mutations are associated with ALS pathogenesis. Therapeutic modulation of angiogenin level and activity in the spinal cord, either by systemic delivery of angiogenin protein or through retrograde transport of ANG-encoding viral particles, may be beneficial for ALS patients.
机译：背景：最近在肌萎缩侧索硬化症（ALS）患者中发现了血管生成素（ANG）基因编码区的错义杂合突变，该突变编码一个14 kDa的血管生成RNase。功能分析表明，这些是功能丧失的突变，这表明血管生成素缺乏与ALS发病机理有关，而增加ANG表达或血管生成素活性可能是ALS治疗的一种新方法。目的：回顾显示血管生成素参与运动神经元生理和功能的证据，并为在ALS治疗中靶向血管生成素提供依据。方法：回顾当前对血管生成素作用机制与ALS遗传学，发病机制和治疗的了解。结论：ANG是第一个功能缺失突变与ALS发病相关的基因。通过全身递送血管生成素蛋白或通过逆向转运编码ANG的病毒颗粒，对脊髓中血管生成素水平和活性的治疗性调节可能对ALS患者有益。

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