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Development of UBA1 Targeted Therapies in VEXAS Syndrome

https://www.coltonconsortium.org/projects/development-of-uba1-targeted-therapies-in-vexas-syndrome/

Building a fluorescence-based cellular screening platform to identify small molecules targeting mutant UBA1, this project addresses an acute unmet need in VEXAS syndrome and lays groundwork for broader rheumatic disease therapies.

The Role and Mechanism of Aberrant Dendritic Cell Function in Autoimmunity

https://www.coltonconsortium.org/projects/the-role-and-mechanism-of-aberrant-dendritic-cell-function-in-autoimmunity/

Identifying a novel molecular regulator of tolerogenic dendritic cell function, this project uncovers how its loss triggers spontaneous multiorgan autoimmunity and exacerbates lupus — revealing a clinically relevant pathway in immune tolerance.

Utilizing RNA Replicons as Immune Modulators for Systemic Lupus Erythematosus

https://www.coltonconsortium.org/projects/utilizing-rna-replicons-as-immune-modulators-for-systemic-lupus-erythematosus/

A self-replicating RNA platform delivers anti-inflammatory cytokines directly to the airways, offering targeted local immune suppression without systemic toxicity — a mechanistically distinct approach to treating lupus lung disease.