Department of Pharmacology, Physiology & Neuroscience, New Jersey Medical School, Rutgers, The State University of New Jersey, Newark, New Jersey, USA.
Adv Neurobiol. 2023;29:391-418. doi: 10.1007/978-3-031-12390-0_13.
The main purpose of this chapter is to summarize the chief findings on ganglioside changes/interactions with some of the neurodegenerative disorders. For the latter we have focused on three diseases that have seen especially intensive study in that regard: Parkinson's, Alzheimer's, and Huntington's diseases. Parkinson's disease (PD) has received the most intensive study with revelation of systemic deficiency of GM1 in brain and all peripheral tissues that have been analyzed to date; this pointed to GM1 replacement as a promising therapy which proved only partially successful when tried for reasons that are discussed. Huntington's disease resembles PD in also manifesting GM1 deficiency, which did, however, respond to GM1 replacement therapy - apparently due to GM1 being administered directly into the brain. Alzheimer's disease was more complex in relation to gangliosides, with b-series (GD1b, GT1b) apparently depressed along with a-series. GM1 administered in brain appeared to induce improvement, but in a limited number of patients. We summarize studies showing why GM1 is of critical importance in neuronal function, and we also briefly point to a few additional neurological disorders in which one or more ganglioside changes have been implicated.
本章的主要目的是总结神经节苷脂变化/与某些神经退行性疾病相互作用的主要发现。对于后者，我们重点关注了三种在这方面得到特别深入研究的疾病：帕金森病、阿尔茨海默病和亨廷顿病。帕金森病（PD）受到了最深入的研究，揭示了脑和所有外周组织中 GM1 的系统性缺乏；这表明 GM1 替代是一种有前途的治疗方法，但由于正在讨论的原因，尝试这种方法仅部分成功。亨廷顿病与 PD 一样，也表现出 GM1 缺乏，但 GM1 替代疗法对其有效——显然是因为 GM1 直接施用于大脑。阿尔茨海默病在与神经节苷脂的关系上更为复杂，b 系列（GD1b、GT1b）与 a 系列一起明显下降。在大脑中给予 GM1 似乎会诱导改善，但在少数患者中。我们总结了表明 GM1 对神经元功能至关重要的研究，我们还简要指出了其他一些神经紊乱疾病，其中涉及一种或多种神经节苷脂变化。
