Sheffield Institute for Translational Neuroscience (SITraN), Department of Neuroscience, University of Sheffield, Sheffield, UK.
Department of Neuroscience, Janssen Pharmaceutica, Beerse, Belgium.
Hum Mol Genet. 2022 Aug 23;31(16):2693-2710. doi: 10.1093/hmg/ddac063.
Hereditary spastic paraplegia type 15 (HSP15) is a neurodegenerative condition caused by the inability to produce SPG15 protein, which leads to lysosomal swelling. However, the link between lysosomal aberrations and neuronal death is poorly explored. To uncover the functional consequences of lysosomal aberrations in disease pathogenesis, we analyze human dermal fibroblasts from HSP15 patients as well as primary cortical neurons derived from an SPG15 knockout (KO) mouse model. We find that SPG15 protein loss induces defective anterograde transport, impaired neurite outgrowth, axonal swelling and reduced autophagic flux in association with the onset of lysosomal abnormalities. Additionally, we observe lipid accumulation within the lysosomal compartment, suggesting that distortions in cellular lipid homeostasis are intertwined with lysosomal alterations. We further demonstrate that SPG15 KO neurons exhibit synaptic dysfunction, accompanied by augmented vulnerability to glutamate-induced excitotoxicity. Overall, our study establishes an intimate link between lysosomal aberrations, lipid metabolism and electrophysiological impairments, suggesting that lysosomal defects are at the core of multiple neurodegenerative disease processes in HSP15.
遗传性痉挛性截瘫 15 型(HSP15)是一种神经退行性疾病,由 SPG15 蛋白产生缺陷引起,导致溶酶体肿胀。然而,溶酶体异常与神经元死亡之间的联系尚未得到充分探索。为了揭示溶酶体异常在疾病发病机制中的功能后果,我们分析了 HSP15 患者的人真皮成纤维细胞以及 SPG15 敲除(KO)小鼠模型来源的原代皮质神经元。我们发现,SPG15 蛋白缺失诱导顺行运输缺陷、突起生长受损、轴突肿胀和自噬流减少,同时伴随着溶酶体异常的发生。此外,我们观察到溶酶体腔内脂质积累,表明细胞脂质稳态的扭曲与溶酶体改变交织在一起。我们进一步证明,SPG15 KO 神经元表现出突触功能障碍,同时对谷氨酸诱导的兴奋性毒性更敏感。总的来说,我们的研究确立了溶酶体异常、脂质代谢和电生理损伤之间的密切联系,表明溶酶体缺陷是 HSP15 中多种神经退行性疾病过程的核心。