Reversing lysosomal dysfunction restores youthful state in aged hematopoietic stem cells

Cell Stem Cell. 2025 Dec 4;32(12):1904-1922.e7. doi: 10.1016/j.stem.2025.10.012. Epub 2025 Nov 24.

Abstract

Aging impairs hematopoietic stem cells (HSCs), driving clonal hematopoiesis, myeloid malignancies, and immune decline. The role of lysosomes in HSC aging-beyond their passive mediation of autophagy-is unclear. We show that lysosomes in aged HSCs are hyperacidic, depleted, damaged, and aberrantly activated. Single-cell transcriptomics and functional analyses reveal that suppression of hyperactivated lysosomes using a vacuolar ATPase (v-ATPase) inhibitor restores lysosomal integrity and metabolic and epigenetic homeostasis in old HSCs. This intervention reduces inflammatory and interferon-driven programs by improving lysosomal processing of mitochondrial DNA and attenuating cyclic GMP-AMP synthase-stimulator of interferon gene (cGAS-STING) signaling. Strikingly, ex vivo lysosomal inhibition boosts old HSCs' in vivo repopulation capacity by over eightfold and improves their self-renewal. Thus, lysosomal dysfunction emerges as a key driver of HSC aging. Targeting hyperactivated lysosomes reinstates a youthful state in old HSCs, offering a promising strategy to restore hematopoietic function in the elderly.

Keywords: MMP; aging; cGas-STING; hematopoietic stem cell; inflammation; interferon; lysosomes; mitochondria; mtDNA; quiescence.

MeSH terms

  • Aging*
  • Animals
  • Cellular Senescence* / drug effects
  • Hematopoietic Stem Cells* / cytology
  • Hematopoietic Stem Cells* / drug effects
  • Hematopoietic Stem Cells* / metabolism
  • Humans
  • Lysosomes* / drug effects
  • Lysosomes* / metabolism
  • Mice
  • Mice, Inbred C57BL
  • Signal Transduction