Genomic Insights and RNA Therapeutics: Advancing Treatment Strategies for Autoimmune Rheumatic Diseases

Affecting over 100 millions individuals globally, autoimmune rheumatic diseases encompass a collection of disorders, for which there is currently no cure.

Irrespective of disease type, the affected organs are infiltrated by auto-reactive immune cells, constantly recirculating between lymphoid organs and injury site via the bloodstream.

A new promising avenune to treat ARDs lies in harnessing the power of RNA therapeutics, which can target any gene of interest.

Until now, translating this potential into tangible therapeutic solutions has been hindered by a lack of data linking disease genetics with cellular function at single-cell resolution.

This project aims to bridge the gap by leveraging data from the world-leading TenK10K study, which includes blood samples from 10000 individuals, from healthy donors and patients with ARDs.

I will integrate cell-type specific genomic data and conduct the largest single-cell expression quantiative locus study to date. This will enable the identification of specific cell types and effector genes that have a causal role in ARD. This research project spans three years and will be co-supervised by Prof.

Powell and Prof. Lappalainen.

The TenK10K cohort enables the study of cells at the finest resolution possible, the resolution required to identify the molecular mechanisms behind diseases in the relevant cell type/s. This project aims to identify new drug targets for RNA therapies in patients with ARDs.