With Funding from the APS Type 1 Foundation, NORD Awards Prestigious Research Grant for Thymus Transplantation
The APS Type 1 Foundation today announces that the National Organization for Rare Disorders (NORD), with funding from the APS Type 1 Foundation, has awarded Dr. Maureen Su, M.D. at the University of North Carolina at Chapel Hill a $100,000 research grant for her study entitled, “Thymus Transplantation for APS1.”
The Su research group is interested in understanding the genetics of autoimmune diseases using both mouse models and patient samples. Their work is highly translational and aims to have direct relevance to human disease. One of their approaches is to study rare Mendelian autoimmunity syndromes in order to determine the contributions of a particular gene to developing autoimmunity. They have focused on Autoimmune Polyendocrinopathy Syndrome Type 1 (APS1 or APECED), a rare condition due to mutations in the Autoimmune Regulator (Aire) gene. They are interested in how Aire promotes tolerance and have utilized both APS1 mouse models and patient samples to understand the disease mechanisms underlying this condition.
“We are grateful for the support of the APS Type 1 Foundation in funding our project to investigate the possibility that thymus organ transplantation may be used to treat patients with APS Type 1. Thymus tranpslantation makes sense from what we now know about the cause of APS Type 1, but much remains to be understood about how this might be translated into a clinically useful therapy. This funding makes it possible for us to define some of the factors that are important for thymus transplants to work in APS Type 1,” said Dr. Maureen Su.
The APS Type 1 Foundation’s mission is to support education and awareness of APS Type 1, an ultra-rare and complex inherited disorder of immune-cell dysfunction with multiple autoimmunities. Since 2008, the Foundation and its founders, in collaboration with NORD’s Research Grant Program, have funded more than $400,000 in restricted research grants focused on APS Type 1. This is the sixth APS Type 1 research grant. The Foundation became a member of NORD in 2016.
“We are proud of the research that has been funded through the financial support of patients and families living with APS Type 1 everyday. Dr. Su’s research is another step that we take toward our goal of realizing a cure for APS Type 1 in the future. We would like to thank NORD’s Medical Advisory Committee for its time and attention in selecting an outstanding research project with Dr. Su and her team’s work at University of North Carolina, Chapel Hill” remarked Todd Talarico, APS Type 1 Foundation President.
The Foundation is sponsoring the Second International Symposium on APS Type 1 from July 13 to 15, 2017,at Stony Brook University, Stony Brook, NY. The symposium will bring together clinicians, researchers, patients and their families. Registration is free at www.apstype1.org.
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