Repository logo
 

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

Accepted version
Peer-reviewed

Change log

Authors

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.

Description

Keywords

Aging, Premature, Animals, DNA Damage, Disease Models, Animal, Female, Genomic Instability, Humans, Hydrazones, Kaplan-Meier Estimate, Lamin Type A, Male, Mice, Mice, Inbred C57BL, Mice, Knockout, N-Terminal Acetyltransferase A, N-Terminal Acetyltransferases, Progeria, Thiazoles

Journal Title

Nat Commun

Conference Name

Journal ISSN

2041-1723
2041-1723

Volume Title

9

Publisher

Springer Science and Business Media LLC
Sponsorship
Cancer Research Uk (None)
Medical Research Council (MR/L019116/1)
Cancer Research UK (18796)
Wellcome Trust (206388/Z/17/Z)
Cancer Research UK (C6946/A24843)
Wellcome Trust (203144/Z/16/Z)