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Current therapies include ERT to compensate for the missing or defective enzyme, and substrate reduction therapy to reduce the accumulation of toxic substances by limiting their production

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Furthermore, BDNF has never entered PD clinical trials, mainly due to poor blood-brain barrier penetration, a short half-life, and the risk of abnormal synaptic plasticity (e.g., epileptiform discharges) with overexpression [62, 63]

Using immunofluorescence and taking advantage of the HA-tag that marks the transformed cells (Methods), we confirmed the presence of this Clu+/HA+ astrocytic glioma cell population in Notch tumors, while Clu was only seen in HA-negative tumor-associated, untransformed astrocytes in the p53 model (Fig

These factors promote the activation of fibroblasts and the excessive production of ECM components