PUBLICATION

Inhibition of PIKfyve Leads to Lysosomal Disorders via Dysregulation of mTOR Signaling

Authors
Xia, J., Wang, H., Zhong, Z., Jiang, J.
ID
ZDB-PUB-240620-10
Date
2024
Source
Cells   13(11): (Journal)
Registered Authors
Keywords
PIKfyve, lysosome, mTOR, macrophage, vacuolation
MeSH Terms
  • Animals
  • Humans
  • Lysosomal Storage Diseases*/genetics
  • Lysosomal Storage Diseases*/metabolism
  • Lysosomal Storage Diseases*/pathology
  • Lysosomes*/metabolism
  • Phosphatidylinositol 3-Kinases*/metabolism
  • Phosphoinositide-3 Kinase Inhibitors/pharmacology
  • Signal Transduction*
  • TOR Serine-Threonine Kinases*/metabolism
  • Zebrafish*
  • Zebrafish Proteins/antagonists & inhibitors
  • Zebrafish Proteins/genetics
  • Zebrafish Proteins/metabolism
PubMed
38891085 Full text @ Cells
Abstract
PIKfyve is an endosomal lipid kinase that synthesizes phosphatidylinositol 3,5-biphosphate from phosphatidylinositol 3-phsphate. Inhibition of PIKfyve activity leads to lysosomal enlargement and cytoplasmic vacuolation, attributed to impaired lysosomal fission processes and homeostasis. However, the precise molecular mechanisms underlying these effects remain a topic of debate. In this study, we present findings from PIKfyve-deficient zebrafish embryos, revealing enlarged macrophages with giant vacuoles reminiscent of lysosomal storage disorders. Treatment with mTOR inhibitors or effective knockout of mTOR partially reverses these abnormalities and extend the lifespan of mutant larvae. Further in vivo and in vitro mechanistic investigations provide evidence that PIKfyve activity is essential for mTOR shutdown during early zebrafish development and in cells cultured under serum-deprived conditions. These findings underscore the critical role of PIKfyve activity in regulating mTOR signaling and suggest potential therapeutic applications of PIKfyve inhibitors for the treatment of lysosomal storage disorders.
Genes / Markers
Figures
Show all Figures
Expression
Phenotype
Mutations / Transgenics
Human Disease / Model
Sequence Targeting Reagents
Fish
Antibodies
Orthology
Engineered Foreign Genes
Mapping