We will create a new dual approach for discovering and developing targeted autoimmune therapeutics. Recent work has highlighted the role of B cells in Neuromyelitis Optica (NMO) disease progression, and autoimmune B cells are a major target for NMO therapeutics. In this project, we will apply a single B cell molecular analysis pipeline to understand the features of autoimmune B cells, and in parallel we will develop a set of new molecular probes designed to silence autoimmune B cells associated with NMO. We will focus our analysis on aquaporin 4, the predominant autoantigen in NMO. We will also apply our molecular-scale technologies to evaluate the ability of these new probes to capture the full set of autoimmune antibodies and autoimmune B cells in NMO patients. This project represents the first step of a broader research program in the guided development and analysis of targeted molecular therapeutics to treat human autoimmune diseases, beginning with NMO.

Public Health Relevance

B cells recognizing aquaporin 4 are a major component of the autoimmune response in patients with Neuromyelitis Optica. This project will develop new strategies to identify and silence anti-aquaporin 4 B cells as new targeted therapeutics.

Agency
National Institute of Health (NIH)
Institute
National Institute of Allergy and Infectious Diseases (NIAID)
Type
Exploratory/Developmental Grants (R21)
Project #
1R21AI144408-01
Application #
9727713
Study Section
Special Emphasis Panel (ZRG1)
Program Officer
Esch, Thomas R
Project Start
2019-01-11
Project End
2020-12-31
Budget Start
2019-01-11
Budget End
2019-12-31
Support Year
1
Fiscal Year
2019
Total Cost
Indirect Cost
Name
University of Kansas Lawrence
Department
Pharmacology
Type
Schools of Pharmacy
DUNS #
076248616
City
Lawrence
State
KS
Country
United States
Zip Code
66045