Tissue inflammation in Mucopolysaccharidoses type II disease: molecular mechanism and therapy

MDBRMPS
Written By Million Dollar Bike Ride

Awardee: Manoj Pandey

Institution: Cincinnati Children's Hospital Medical Center

Award Amount: $64,485

Funding Period: February 1, 2021 - January 31, 2022

Summary:

Due to iduronate 2-sulfatase enzyme deficiency, excess tissue accumulation of glycosaminoglycans (GAGs) lead to the chronic tissue inflammation in Mucopolysaccharidoses type II (MPSII) patients. The mechanisms underlying GAGs-mediated chronic tissue inflammation is remain elusive. Our preliminary data identified GAGs-induced complement activation as one of the main driver of immune inflammation that sparks tissue inflammation in MPSII. Proposed studies will now test if targeting complement activation directly in MPSII-mouse model and human cells could stop and/or slowdown the tissue inflammation. Additionally, complement activation at several steps and/or their signature cytokines could recognize as a novel biomarker for human MPSII.

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