A Lipophilic 4-Phenylbutyric Acid Derivative That Prevents Aggregation and Retention of Misfolded Proteins.
Animals
Cell Adhesion Molecules, Neuronal
/ chemistry
Cell Survival
/ drug effects
HEK293 Cells
Humans
Membrane Proteins
/ chemistry
Mutagenesis, Site-Directed
Nerve Tissue Proteins
/ chemistry
PC12 Cells
Phenylbutyrates
/ chemistry
Protein Aggregates
/ drug effects
Protein Folding
Proteins
/ chemistry
Rats
ER stress
aggregation
chaperones
lipophilicity
protein folding
Journal
Chemistry (Weinheim an der Bergstrasse, Germany)
ISSN: 1521-3765
Titre abrégé: Chemistry
Pays: Germany
ID NLM: 9513783
Informations de publication
Date de publication:
06 Feb 2020
06 Feb 2020
Historique:
received:
18
09
2019
revised:
17
11
2019
pubmed:
28
11
2019
medline:
14
2
2020
entrez:
28
11
2019
Statut:
ppublish
Résumé
Chemical chaperones prevent protein aggregation. However, the use of chemical chaperones as drugs against diseases due to protein aggregation is limited by the very high active concentrations (mm range) required to mediate their effect. One of the most common chemical chaperones is 4-phenylbutyric acid (4-PBA). Despite its unfavorable pharmacokinetic properties, 4-PBA was approved as a drug to treat ornithine cycle diseases. Here, we report that 2-isopropyl-4-phenylbutanoic acid (5) has been found to be 2-10-fold more effective than 4-PBA in several in vitro models of protein aggregation. Importantly, compound 5 reduced the secretion rate of autism-linked Arg451Cys Neuroligin3 (R451C NLGN3).
Identifiants
pubmed: 31773792
doi: 10.1002/chem.201904292
doi:
Substances chimiques
Cell Adhesion Molecules, Neuronal
0
Membrane Proteins
0
Nerve Tissue Proteins
0
Phenylbutyrates
0
Protein Aggregates
0
Proteins
0
neuroligin 3
0
4-phenylbutyric acid
7WY7YBI87E
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
1834-1845Subventions
Organisme : Israel Ministry of Science and the Italian Ministry of Foreign Affairs
ID : 3-13325
Organisme : NAAMAT, for the Edelson Foundation prize for outstanding women researchers
Organisme : Navon fellowship for PhD students, awarded by the Israel Ministry of Science, Technology and Space.
Organisme : German-Israeli Foundation for Scientific Research and Development
ID : I-1410-201.9/2017
Organisme : the Israel Ministry of Science and the Italian Ministry of Foreign Affairs
ID : 3-13325
Organisme : the Navon fellowship for PhD students, awarded by the Israel Ministry of Science, Technology and Space.
Informations de copyright
© 2019 Wiley-VCH Verlag GmbH & Co. KGaA, Weinheim.
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