METHOD AND DRUG FOR TREATING NEURONAL CEROID LIPOFUSCINOSIS

Abstract: a method for treating a disease associated with cell autophagy functional deficiency and/or lysosomal functional deficiency, such as neurodegenerative diseases, by inhibiting keap1 activity and/or enhancing the expression of nuclear erythroid 2-related factor 2 (nrf2) and/or the downstream genes thereof. the expression of nrf2 and/or the downstream genes thereof is enhanced with a keap1 inhibitor, a keap1 gene knockdown, and/or an nrf2 activator. the method and product can increase the acidic environment in the lysosomes of neurons, increase the enzymatic activity of cathepsins, decrease the abnormal storage of proteins, protect mitochondrial homeostasis in neurons, and increase the capability of mitochondria to generate atp.

IPC:

CPC: A61K31/57,