2023年 新着論文 70 蛋白質発現分野から論文が発表されました

Lysoglycosphingolipids have the ability to induce cell death through direct PI3K inhibition

J Neurochem. 2023 Dec;167(6):753-765. doi: 10.1111/jnc.16012. Epub 2023 Nov 17.

Authors

Ryosuke Watanabe  1 Daisuke Tsuji  1   2 Hiroki Tanaka  1 Michael Shintaro Uno  1 Yukiya Ohnishi  1 Shindai Kitaguchi  1 Tsuyoshi Matsugu  1 Ryuto Nakae  1 Hiromi Teramoto  1 Kei Yamamoto  3 Yasuo Shinohara  4   5 Takatsugu Hirokawa  6 Nozomu Okino  7 Makoto Ito  7 Kohji Itoh  1

Affiliations

  • 1 Department of Medicinal Biotechnology, Graduate school of Pharmaceutical Science, Tokushima University, Tokushima, Japan.
  • 2 Department of Pharmacy, Faculty of Pharmacy, Yasuda Women’s University, Hiroshima, Japan.
  • 3 Faculty of Bioscience and Bioindustry, Tokushima University, Tokushima, Japan.
  • 4 Institute for Genome Research, Institute of Advanced Medical Sciences, Tokushima University, Tokushima, Japan.
  • 5 Graduate School of Pharmaceutical Sciences, Tokushima University, Tokushima, Japan.
  • 6 Molecular Profiling Research Center for Drug Discovery (molprof), National Institute of Advanced Industrial Science and Technology (AIST), Tokyo, Japan.
  • 7 Department of Bioscience and Biotechnology, Graduate School of Bioresource and Bioenvironmental Sciences, Kyushu University, Fukuoka, Japan.

Abstract

Sphingolipidoses are inherited metabolic disorders associated with glycosphingolipids accumulation, neurodegeneration, and neuroinflammation leading to severe neurological symptoms. Lysoglycosphingolipids (lysoGSLs), also known to accumulate in the tissues of sphingolipidosis patients, exhibit cytotoxicity. LysoGSLs are the possible pathogenic cause, but the mechanisms are still unknown in detail. Here, we first show that lysoGSLs are potential inhibitors of phosphoinositide 3-kinase (PI3K) to reduce cell survival signaling. We found that phosphorylated Akt was commonly reduced in fibroblasts from patients with sphingolipidoses, including GM1/GM2 gangliosidoses and Gaucher’s disease, suggesting the contribution of lysoGSLs to the pathogenesis. LysoGSLs caused cell death and decreased the level of phosphorylated Akt as in the patient fibroblasts. Extracellularly administered lysoGM1 permeated the cell membrane to diffusely distribute in the cytoplasm. LysoGM1 and lysoGM2 also inhibited the production of phosphatidylinositol-(3,4,5)-triphosphate and the translocation of Akt from the cytoplasm to the plasma membrane. We also predicted that lysoGSLs could directly bind to the catalytic domain of PI3K by in silico docking study, suggesting that lysoGSLs could inhibit PI3K by directly interacting with PI3K in the cytoplasm. Furthermore, we revealed that the increment of lysoGSLs amounts in the brain of sphingolipidosis model mice correlated with the neurodegenerative progression. Our findings suggest that the down-regulation of PI3K/Akt signaling by direct interaction of lysoGSLs with PI3K in the brains is a neurodegenerative mechanism in sphingolipidoses. Moreover, we could propose the intracellular PI3K activation or inhibition of lysoGSLs biosynthesis as novel therapeutic approaches for sphingolipidoses because lysoGSLs should be cell death mediators by directly inhibiting PI3K, especially in neurons.

Keywords: PI3K; cell death; glycosphingolipids; lipid mediator; sphingolipidoses.

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