Targeting of NAT10 enhances healthspan in a mouse model of human accelerated aging syndrome

Nat Commun. 2018 Apr 27;9(1):1700. doi: 10.1038/s41467-018-03770-3.

Abstract

Hutchinson-Gilford Progeria Syndrome (HGPS) is a rare, but devastating genetic disease characterized by segmental premature aging, with cardiovascular disease being the main cause of death. Cells from HGPS patients accumulate progerin, a permanently farnesylated, toxic form of Lamin A, disrupting the nuclear shape and chromatin organization, leading to DNA-damage accumulation and senescence. Therapeutic approaches targeting farnesylation or aiming to reduce progerin levels have provided only partial health improvements. Recently, we identified Remodelin, a small-molecule agent that leads to amelioration of HGPS cellular defects through inhibition of the enzyme N-acetyltransferase 10 (NAT10). Here, we show the preclinical data demonstrating that targeting NAT10 in vivo, either via chemical inhibition or genetic depletion, significantly enhances the healthspan in a Lmna G609G HGPS mouse model. Collectively, the data provided here highlights NAT10 as a potential therapeutic target for HGPS.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Aging, Premature / drug therapy*
  • Aging, Premature / genetics
  • Aging, Premature / mortality
  • Aging, Premature / pathology
  • Animals
  • DNA Damage / drug effects
  • DNA Damage / genetics
  • Disease Models, Animal
  • Female
  • Genomic Instability / drug effects*
  • Genomic Instability / genetics
  • Humans
  • Hydrazones / pharmacology*
  • Hydrazones / therapeutic use
  • Kaplan-Meier Estimate
  • Lamin Type A / genetics
  • Male
  • Mice
  • Mice, Inbred C57BL
  • Mice, Knockout
  • N-Terminal Acetyltransferase A / antagonists & inhibitors*
  • N-Terminal Acetyltransferase A / genetics
  • N-Terminal Acetyltransferase A / metabolism
  • N-Terminal Acetyltransferases
  • Progeria / drug therapy*
  • Progeria / genetics
  • Progeria / mortality
  • Progeria / pathology
  • Thiazoles / pharmacology*
  • Thiazoles / therapeutic use

Substances

  • 4-(4-cyanophenyl)-2-(2-cyclopentylidenehydrazinyl)thiazole
  • Hydrazones
  • Lamin Type A
  • Lmna protein, mouse
  • Thiazoles
  • N-Terminal Acetyltransferase A
  • N-Terminal Acetyltransferases
  • Nat10 protein, mouse