Induction of PAX5 expression as a potential therapeutic approach for Mucolipidosis IV

The project aims to induce PAX5 expression in the brain to mitigate pathological inflammatory responses that are observed in the brain of Mucolipidosis IV (MLIV) mouse model. PAX5 expression in neuronal and glial cells appear to reduce the expression of genes involved in inflammation. Transcriptomic (RNA sequencing) analysis of neuronal and glial cells induced to express PAX5 showed down-regulation of inflammatory genes that could minimize or stop the pathological contribution of microglial activation caused by the disease.