Article

Small-molecule Activation of TFEB Alleviates Niemann-Pick Disease Type C via Promoting Lysosomal Exocytosis and Biogenesis

2024-10-17

Abstract excerpt

Niemann–Pick disease type C (NPC) is a devastating lysosomal storage disease characterized by abnormal cholesterol accumulation in lysosomes. Currently, there is no treatment for NPC. Transcription factor EB (TFEB), a member of the microphthalmia transcription factors (MiTF), has emerged as a master regulator of lysosomal function and promoted the clearance of substrates stored in cells. However, it is not known w...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work: 68c6f6d6-599c-54fc-9860-4423210a2831
DOI: 10.1101/2024.10.15.616556

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

Small-molecule Activation of TFEB Alleviates Niemann-Pick Disease Type C via Promoting Lysosomal Exocytosis and BiogenesisDOI 10.1101/2024.10.15.616556Read

The regulatory mechanism and therapeutic potential of transcription factor <scp>EB</scp> in neurodegenerative diseasesSemantic proximity Read

Niemann-Pick Disease Type C: from molecule to clinic.Semantic proximity Read

TFEB and TFE3 have cell-type specific expression in the brain and divergent roles in neuronsSemantic proximity Read

The unique case of the Niemann-Pick type C cholesterol storage disorder.Semantic proximity Read

Transcription factor EB: from master coordinator of lysosomal pathways to candidate therapeutic target in degenerative storage diseasesSemantic proximity Read

Disease-associated mutations in Niemann-Pick type C1 alter ER calcium signaling and neuronal plasticitySemantic proximity Read

TFEB Biology and Agonists at a GlanceSemantic proximity Read

Niemann-Pick type C disease: molecular mechanisms and potential therapeutic approaches.Semantic proximity Read

Novel Insight into Functions of Transcription Factor EB (TFEB) in Alzheimer’s Disease and Parkinson’s DiseaseSemantic proximity Read

The pathogenesis of Niemann–Pick type C disease: a role for autophagy?Semantic proximity Read

Overview of clinical, molecular, and therapeutic features of Niemann-Pick disease (types A, B, and C): Focus on therapeutic approaches.Semantic proximity Read

The Role and Regulatory Mechanism of Transcription Factor EB in Health and DiseasesSemantic proximity Read