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Single-Cell RNA sequencing identifies NAMPT as a potential therapeutic target in autoimmune uveitis
Yingying Wen1, Lei Zhu2, Zhaohao Huang3
1Bengbu Medical University, Bengbu 233000, China.
Journal of Advanced Research
|December 31, 2025
Summary
Inhibiting NAMPT, an enzyme involved in immune regulation, effectively treats autoimmune uveitis (AU) by restoring T cell balance. This finding identifies NAMPT as a promising therapeutic target for AU and related inflammatory eye diseases.
Area of Science:
- Immunology
- Ophthalmology
- Molecular Biology
Background:
- Autoimmune uveitis (AU) causes irreversible vision loss, necessitating new treatments due to current therapy limitations.
- Nicotinamide phosphoribosyltransferase (NAMPT) has known immunoregulatory roles, but its specific involvement in AU was previously unclear.
Purpose of the Study:
- To investigate the therapeutic potential of targeting NAMPT in experimental autoimmune uveitis (EAU).
- To elucidate the underlying mechanisms of NAMPT's action on immune cells in AU.
Main Methods:
- Single-cell RNA sequencing (scRNA-seq) was used on mouse lymph nodes and human peripheral blood mononuclear cells (PBMCs).
- NAMPT inhibition was assessed in mouse models of AU and human Vogt-Koyanagi-Harada (VKH) disease.
- Flow cytometry and adoptive transfer experiments validated findings.
Main Results:
- NAMPT inhibition significantly reduced AU severity and reversed disease-associated immune cell profiles.
- Targeting NAMPT modulated the balance of T helper 17 (Th17) and regulatory T cells (Tregs) by downregulating Hif1α.
- Elevated NAMPT expression in human VKH patients correlated with increased pro-inflammatory T cells and decreased Tregs.
Conclusions:
- NAMPT inhibition restores Teff/Treg cell balance via the NAMPT-Hif1α axis in CD4+ T cells, alleviating AU.
- NAMPT represents a potential therapeutic target for autoimmune uveitis.
