在慢性实验性过敏性脑脊髓炎中,成人少突胶质细胞中半乳糖基甘油糖苷酶的缺乏会加重神经功能缺损并缩短存活时间。

IF 12.1 1区 医学 Q1 BIOTECHNOLOGY & APPLIED MICROBIOLOGY Molecular Therapy Pub Date : 2024-09-04 Epub Date: 2024-06-26 DOI:10.1016/j.ymthe.2024.06.035
Natalia Saldivia, Gregory Heller, Diego Zelada, Jason Whitehair, Nikhil Venkat, Ashna Konjeti, Reina Savitzky, Shayla Samano, Daniel Simchuk, Richard van Breemen, Maria I Givogri, Ernesto R Bongarzone
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引用次数: 0

摘要

半乳糖基神经酰胺酶(GALC)是一种无处不在的溶酶体酶,对哺乳动物神经系统在出生后早期的正确髓鞘化至关重要。然而,GALC 缺乏对成年大脑的生理影响仍然未知。在这项研究中,我们发现在髓鞘化后少突胶质细胞中存在条件性 GALC 活性消减的小鼠在受到慢性实验性过敏性脑脊髓炎(EAE)的挑战时会出现致命的致敏现象,这与在没有受到 EAE 挑战的 GALC 消减小鼠中观察到的非致命性髓鞘化障碍形成了鲜明对比。从机理上讲,我们发现了一种强烈的炎症性脱髓鞘而无再髓鞘化的现象,以及溶酶体和自噬体融合受损,髓鞘碎屑随着依赖于 TFEB 的溶酶体自噬体通量的增加而积聚。这些结果表明,GALC 缺乏的生理影响受到细胞环境(少突胶质细胞与整体表达)、炎症的存在以及发生的发育时间(髓鞘化前与髓鞘化后)的很大影响。我们的结论是,成体少突胶质细胞中 GALC 的表达对于维持成体中枢髓鞘和降低对其他脱髓鞘损伤的脆弱性至关重要。
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Deficiency of galactosyl-ceramidase in adult oligodendrocytes worsens disease severity during chronic experimental allergic encephalomyelitis.

Galactosyl-ceramidase (GALC) is a ubiquitous lysosomal enzyme crucial for the correct myelination of the mammalian nervous system during early postnatal development. However, the physiological consequence of GALC deficiency in the adult brain remains unknown. In this study, we found that mice with conditional ablation of GALC activity in post-myelinating oligodendrocytes were lethally sensitized when challenged with chronic experimental allergic encephalomyelitis (EAE), in contrast with the non-lethal dysmyelination observed in Galc-ablated mice without the EAE challenge. Mechanistically, we found strong inflammatory demyelination without remyelination and an impaired fusion of lysosomes and autophagosomes with accumulation of myelin debris after a transcription factor EB-dependent increase in the lysosomal autophagosome flux. These results indicate that the physiological impact of GALC deficiency is highly influenced by the cell context (oligodendroglial vs. global expression), the presence of inflammation, and the developmental time when it happens (pre-myelination vs. post-myelination). We conclude that Galc expression in adult oligodendrocytes is crucial for the maintenance of adult central myelin and to decrease vulnerability to additional demyelinating insults.

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来源期刊
Molecular Therapy
Molecular Therapy 医学-生物工程与应用微生物
CiteScore
19.20
自引率
3.20%
发文量
357
审稿时长
3 months
期刊介绍: Molecular Therapy is the leading journal for research in gene transfer, vector development, stem cell manipulation, and therapeutic interventions. It covers a broad spectrum of topics including genetic and acquired disease correction, vaccine development, pre-clinical validation, safety/efficacy studies, and clinical trials. With a focus on advancing genetics, medicine, and biotechnology, Molecular Therapy publishes peer-reviewed research, reviews, and commentaries to showcase the latest advancements in the field. With an impressive impact factor of 12.4 in 2022, it continues to attract top-tier contributions.
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