Correction: Corrigendum: mTORC1-independent TFEB activation via Akt inhibition promotes cellular clearance in neurodegenerative storage diseases

Palmieri, Michela; Pal, Rituraj; Nelvagal, Hemanth R.; Lotfi, Parisa; Stinnett, Gary; Seymour, Michelle L.; Chaudhury, Arindam; Bajaj, Lakshya; Bondar, Vitaliy V.; Bremner, Laura; Saleem, Usama; Tse, Dennis Y.; Sanagasetti, Deepthi; Wu, Samuel M.; Neilson, Joel R.; Pereira, Fred A.; Pautler, Robia G.; Rodney, George G.; Cooper, Jonathan D.; Sardiello, Marco

doi:10.1038/ncomms15793

Cited by 26 publications

(15 citation statements)

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“…Intraperitoneal injection of MK2206 leads to inhibition of Akt activity and to TFEB nuclear translocation in a mouse brain, which in turn promotes the up-regulation of lysosomal and autophagic genes. It provides the evidence of enhanced pharmacological activation of TFEB for autophagy-lysosomal pathway in vitro and in vivo [97].…”

Section: Clinical Trials and Preclinical Trials Of Tfeb Agonistsmentioning

confidence: 93%

TFEB Biology and Agonists at a Glance

Chen

Dai

Liu

et al. 2021

Cells

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Autophagy is a critical regulator of cellular survival, differentiation, development, and homeostasis, dysregulation of which is associated with diverse diseases including cancer and neurodegenerative diseases. Transcription factor EB (TFEB), a master transcriptional regulator of autophagy and lysosome, can enhance autophagic and lysosomal biogenesis and function. TFEB has attracted a lot of attention owing to its ability to induce the intracellular clearance of pathogenic factors in a variety of disease models, suggesting that novel therapeutic strategies could be based on the modulation of TFEB activity. Therefore, TFEB agonists are a promising strategy to ameliorate diseases implicated with autophagy dysfunction. Recently, several TFEB agonists have been identified and preclinical or clinical trials are applied. In this review, we present an overview of the latest research on TFEB biology and TFEB agonists.

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Section: Clinical Trials and Preclinical Trials Of Tfeb Agonistsmentioning

confidence: 93%

TFEB Biology and Agonists at a Glance

Chen

Dai

Liu

et al. 2021

Cells

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“…Correspondingly, some studies indicate that EBV uses autophagic machinery for transport and cytosolic maturation [ 139 , 141 ]. The presence of EBV BFRF1 is crucial for the early steps of autophagy and blocks the final proteolysis in the EBV life cycle [ 140 ].…”

Section: Ebv Putatively Hijacks the Endogenous Transporting/clearance Pathway For Its Maturationmentioning

confidence: 99%

Conquering the Nuclear Envelope Barriers by EBV Lytic Replication

Lee

Chen

2021

Viruses

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The nuclear envelope (NE) of eukaryotic cells has a highly structural architecture, comprising double lipid-bilayer membranes, nuclear pore complexes, and an underlying nuclear lamina network. The NE structure is held in place through the membrane-bound LINC (linker of nucleoskeleton and cytoskeleton) complex, spanning the inner and outer nuclear membranes. The NE functions as a barrier between the nucleus and cytoplasm and as a transverse scaffold for various cellular processes. Epstein–Barr virus (EBV) is a human pathogen that infects most of the world’s population and is associated with several well-known malignancies. Within the nucleus, the replicated viral DNA is packaged into capsids, which subsequently egress from the nucleus into the cytoplasm for tegumentation and final envelopment. There is increasing evidence that viral lytic gene expression or replication contributes to the pathogenesis of EBV. Various EBV lytic proteins regulate and modulate the nuclear envelope structure in different ways, especially the viral BGLF4 kinase and the nuclear egress complex BFRF1/BFRF2. From the aspects of nuclear membrane structure, viral components, and fundamental nucleocytoplasmic transport controls, this review summarizes our findings and recently updated information on NE structure modification and NE-related cellular processes mediated by EBV.

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“…However, the long-term effects of the regulation of the proteostasis cannot be predicted. The link between autophagy impairment and lysosomal disorders has led to novel approaches to modulate cellular clearance, which has been investigated in preclinical studies (Palmieri et al, 2017), but the translation to clinical trials is still premature.…”

Section: Proteostasis Regulatorsmentioning

confidence: 99%

CNS-Targeting Therapies for Lysosomal Storage Diseases: Current Advances and Challenges

Edelmann

Maegawa

2020

Front. Mol. Biosci.

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During the past decades, several therapeutic approaches have been developed and made rapidly available for many patients afflicted with lysosomal storage disorders (LSDs), inborn organelle disorders with broad clinical manifestations secondary to the progressive accumulation of undegraded macromolecules within lysosomes. These conditions are individually rare, but, collectively, their incidence ranges from 1 in 2,315 to 7,700 live-births. Most LSDs are manifested by neurological symptoms or signs, including developmental delay, seizures, acroparesthesia, motor weakness, and extrapyramidal signs. The chronic and later-onset clinical forms are at one end of the continuum spectrum and are characterized by a subtle and slow progression of neurological symptoms. Due to its inherent physiological properties, unfortunately, the blood-brain barrier (BBB) constitutes a significant obstacle for current and upcoming therapies to achieve the central nervous system (CNS) and treat neurological problems so prevalent in these conditions. To circumvent this limitation, several strategies have been developed to make the therapeutic agent achieve the CNS. This narrative will provide an overview of current therapeutic strategies under development to permeate the BBB, and address and unmet need for treatment of the progressive neurological manifestations, which are so prevalent in these inherited lysosomal disorders.

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Correction: Corrigendum: mTORC1-independent TFEB activation via Akt inhibition promotes cellular clearance in neurodegenerative storage diseases

Cited by 26 publications

References 1 publication

TFEB Biology and Agonists at a Glance

TFEB Biology and Agonists at a Glance

Conquering the Nuclear Envelope Barriers by EBV Lytic Replication

CNS-Targeting Therapies for Lysosomal Storage Diseases: Current Advances and Challenges

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