Journal
NATURE COMMUNICATIONS
Volume 9, Issue -, Pages -Publisher
NATURE RESEARCH
DOI: 10.1038/s41467-018-03770-3
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Funding
- Cancer Research UK (CRUK) program grant [C6/A18796]
- Wellcome Trust Investigator Award [206388/Z/17/Z]
- CRUK [C6946/A24843]
- Wellcome Trust [WT203144]
- Project Grant from the Medical Research Council UK [MR/L019116/1]
- CRUK
- Wellcome Trust
- European Research Council [647973]
- Emergence Ville de Paris Program
- European Research Council grant DDREAM
- MRC [MR/L019116/1, MC_U105181010] Funding Source: UKRI
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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.
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