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.

本项目旨在诱导大脑中PAX5的表达，以减轻黏脂贮积症IV型（Mucolipidosis IV, MLIV）小鼠模型大脑中观察到的病理性炎症反应。神经元与神经胶质细胞内的PAX5表达，可降低炎症相关基因的表达水平。对经诱导表达PAX5的神经元及神经胶质细胞开展转录组（RNA测序）分析后发现，炎症相关基因出现下调，这可减轻乃至阻断该疾病引发的小胶质细胞活化所带来的病理性影响。