Tissue Profiling of the Th17 Gene Activity in AS
Concept
Ankylosing Spondylitis (AS) is a chronic immune-mediated disease that affects various musculoskeletal structures and extra-articular organs, such as the skin, the gut and the eye. In AS, Th17 cells drive inflammation and tissue damage. Although targeting Th17 cells represents an effective treatment strategy, over half of patients fail to respond, and this class of drugs does not provides benefit on the AS-associated colitis. The plan is to perform single cell sequencing of Th17 cells from blood and 3 common sites of AS inflammation: peripheral joint, gut mucosa and psoriatic skin.
Facts and Figures
D Simone
Università della Campania Vanvitelli
davide.simone@unicampania.it
Meet the Team
Università della Campania Vanvitelli
Objectives
Ankylosing Spondylitis (AS) is a chronic rheumatic disease, in which an altered immune system causes excessive inflammation in the joints, the spine, the skin and the gut. Immune cells are able to adapt to the surroundings by switching their genes on and off, and this makes the available medications not always effective on all the organ manifestations of AS. The aim of this research is to provide an in depth study of a class of immune cells called Th17, isolated from the blood and the organs of AS patients, using a novel high-resolution technology called single cell sequencing. This technique is able to show how these cells modulate their genome during a disease flare in each organ, and to reveal novel targets for effective treatments for AS.
Goals/Milestones
- 6 months sample processing and RNA sequencing in batches. Incl. 4 months for patient recruitment and sample collection.
- 6 months of computational analysis.
Patient Voice
For the initial gene sequencing, 9 patients, 3 for each of 3 typical manifestation of the disease (joint, intestine, skin) will be recruited. AS is a severe, debilitating condition, typically diagnosed before the age of 40, which carries life-long impact. There is also considerable need for new, more effective drugs, because a number of patients do not respond to the available treatments, which are often very expensive.