Understanding the role of inflammation in MPSIIIA disease to identify therapeutic approaches for older patients without treatment options
Awardee: Brian Bigger
Institution: University of Manchester
Grant Amount: $64,015
Funding Period: February 1, 2022 - January 31, 2023
Summary:
Many older patients with mucopolysaccharide diseases are ineligible for new treatment trials, and those with brain disease, such as type IIIA, have no current treatments available. To address this gap, we want to explore in the mouse model of MPSIIIA, the observed decline in cognition that follows repeated rounds of viral infection in patients and has been described in many dementias. Our aim is to first see if these effects can be recapitulated in the MPSIIIA mouse model, and later to develop novel immunotherapies to treat it. There are several drugs already available that may be re-purposable to this end, potentially offering rapid relief for MPS patients from behavioural and cognitive aspects of disease