LAUSR

Proteostasis Regulators in Cystic Fibrosis: Current Development and Future Perspectives

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1 Published in 2022 at "Journal of Medicinal Chemistry"

DOI: 10.1021/acs.jmedchem.1c01897

Abstract: In cystic fibrosis (CF), the deletion of phenylalanine 508 (F508del) in the CF transmembrane conductance regulator (CFTR) leads to misfolding and premature degradation of the mutant protein. These defects can be targeted with pharmacological agents… read more here.

Keywords: proteostasis regulators; proteostasis; fibrosis current; regulators cystic ... See more keywords

Proteostasis regulators modulate proteasomal activity and gene expression to attenuate multiple phenotypes in Fabry disease

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0 Published in 2020 at "Biochemical Journal"

DOI: 10.1042/bcj20190513

Abstract: The lysosomal storage disorder Fabry disease is characterized by a deficiency of the lysosomal enzyme α-Galactosidase A. The observation that missense variants in the encoding GLA gene often lead to structural destabilization, endoplasmic reticulum retention… read more here.

Keywords: fabry disease; proteostasis; gene expression; activity ... See more keywords

Floxuridine supports UPS independent of germline signaling and proteostasis regulators via involvement of detoxification in C. elegans

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0 Published in 2024 at "PLOS Genetics"

DOI: 10.1371/journal.pgen.1011371

Abstract: The ubiquitin-proteasome system (UPS) is critical for maintaining proteostasis, influencing stress resilience, lifespan, and thermal adaptability in organisms. In Caenorhabditis elegans, specific proteasome subunits and activators, such as RPN-6, PBS-6, and PSME-3, are associated with… read more here.

Keywords: floxuridine supports; ups independent; proteostasis; proteostasis regulators ... See more keywords

Pharmaceutical Chaperones and Proteostasis Regulators in the Therapy of Lysosomal Storage Disorders: Current Perspective and Future Promises

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0 Published in 2017 at "Frontiers in Pharmacology"

DOI: 10.3389/fphar.2017.00448

Abstract: Different approaches have been utilized or proposed for the treatment of lysosomal storage disorders (LSDs) including enzyme replacement and hematopoietic stem cell transplant therapies, both aiming to compensate for the enzymatic loss of the underlying… read more here.

Keywords: proteostasis regulators; lysosomal storage; proteostasis; pharmaceutical chaperones ... See more keywords