Topic: Transcription factor EB

Lysosomal storage disorders (LSD) are a group of rare inherited metabolic disorders caused by defects in lysosomal function. Among these, Mucopolysaccharidoses (MPS) are characterized by the loss of function of lysosomal enzymes responsible for the degradation of glycosaminoglycans (GAGs). This defect leads to the production, at the cellular level, of secondary storages comprised of undegraded proteins. In some of these syndromes, this translates into a neurodegenerative phenotype with pediatric dementia. Promoting the degradation of secondary storages is one of the most promising therapeutic

Trehalose is a natural disaccharide formed by a 1,1-glycosidic bond between two α-glucose units, widely used in food industry due to its peculiar chemical properties. In the last 20 years the involuntary trehalose intake is exponentially increased, with unknown long-term effects on the population. Furthermore, circulating trehalose is able to reach the CNS. Importantly, trehalose induces autophagy in an mTORC1-independent manner activating the transcription factor EB (TFEB), the master transcriptional regulator of autophagy-lysosomal biogenesis. Accordingly, several studies indicated that treh

Alzheimer’s disease (AD) presents a significant challenge to therapeutic interventions, necessitating the exploration of innovative strategies to manage its complex pathology. This study explores the relationship between autophagy and Transcription factor EB (TFEB) as a potential avenue for AD treatment. We demonstrate the therapeutic potential of TFEB in mitigating AD pathology by enhancing autophagy. Our strategy employs exosomes as vehicles for the targeted and efficient delivery of TFEB to affected cells. Exosomes, small extracellular vesicles, are recognized for their ability to cross bio