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4-Phenylbutyric Acid (4-PBA) Derivatives Prevent SOD1 Amyloid Aggregation In Vitro with No Effect on Disease Progression in SOD1-ALS Mice.
Alfahel L, Argueti-Ostrovsky S, Barel S, Ali Saleh M, Kahn J, Azoulay-Ginsburg S, Rothstein A, Ebbinghaus S, Gruzman A, Israelson A. Alfahel L, et al. Int J Mol Sci. 2022 Aug 20;23(16):9403. doi: 10.3390/ijms23169403. Int J Mol Sci. 2022. PMID: 36012668 Free PMC article.
In the present study, we investigated the potential therapeutic effect of C4 and C5, two derivatives of the chemical chaperone 4-phenylbutyric acid (4-PBA). By combining in vivo and in vitro techniques, we show that, although C4 and C5 successfully inhibited amyloid …
In the present study, we investigated the potential therapeutic effect of C4 and C5, two derivatives of the chemical chaperone 4-phenylbu …
Intracellular spatially-targeted chemical chaperones increase native state stability of mutant SOD1 barrel.
Ribeiro SS, Gnutt D, Azoulay-Ginsburg S, Fetahaj Z, Spurlock E, Lindner F, Kuz D, Cohen-Erez Y, Rapaport H, Israelson A, Gruzman AL, Ebbinghaus S. Ribeiro SS, et al. Biol Chem. 2023 Aug 9;404(10):909-930. doi: 10.1515/hsz-2023-0198. Print 2023 Sep 26. Biol Chem. 2023. PMID: 37555646
We found that compound ER-15 specifically increased the native state stability of ER-SOD1(bar)-A4V, while scaffold compound FDA-approved 4-phenylbutyric acid (PBA) decreased it. Furthermore, our results suggested that ER15 mechanism of action is distinct from that o …
We found that compound ER-15 specifically increased the native state stability of ER-SOD1(bar)-A4V, while scaffold compound FDA-approved 4- …
A missense mutation in the proprotein convertase gene furinb causes hepatic cystogenesis during liver development in zebrafish.
Ellis JL, Evason KJ, Zhang C, Fourman MN, Liu J, Ninov N, Delous M, Vanhollebeke B, Fiddes I, Otis JP, Houvras Y, Farber SA, Xu X, Lin X, Stainier DYR, Yin C. Ellis JL, et al. Hepatol Commun. 2022 Nov;6(11):3083-3097. doi: 10.1002/hep4.2038. Epub 2022 Aug 26. Hepatol Commun. 2022. PMID: 36017776 Free PMC article.
The cystic phenotype could be suppressed by treatment with the ER stress inhibitor 4-phenylbutyric acid and exacerbated by treatment with the ER stress inducer tunicamycin. The mutant liver also exhibited increased mammalian target of rapamycin (mTOR) signaling. ...
The cystic phenotype could be suppressed by treatment with the ER stress inhibitor 4-phenylbutyric acid and exacerbated by tre …
Thymoquinone inhibited the chondrogenic differentiation of tendon-derived stem cells caused by tendon injury.
Tu YJ, Liu YQ, Pan YY, Cai HY, Liu C. Tu YJ, et al. World J Stem Cells. 2025 Nov 26;17(11):112393. doi: 10.4252/wjsc.v17.i11.112393. World J Stem Cells. 2025. PMID: 41356382 Free PMC article.

Furthermore, TQ diminished the chondrogenic differentiation of TDSCs, as evidenced by decreased expression of collagen II (4.80 0.47 vs 1.38 0.28, P < 0.001), aggrecan (2.83 0.26 vs 1.44 0.19, P < 0.001), and SOX9 (4.13 0.46 vs 1.26 0.25, P < 0.001), effects

…
Furthermore, TQ diminished the chondrogenic differentiation of TDSCs, as evidenced by decreased expression of collagen II (4.80 0.47 vs 1. …
Chemical chaperones targeted to the endoplasmic reticulum (ER) and lysosome prevented neurodegeneration in a C9orf72 repeat expansion drosophila amyotrophic lateral sclerosis (ALS) model.
Azoulay-Ginsburg S, Di Salvio M, Weitman M, Afri M, Ribeiro S, Ebbinghaus S, Cestra G, Gruzman A. Azoulay-Ginsburg S, et al. Pharmacol Rep. 2021 Apr;73(2):536-550. doi: 10.1007/s43440-021-00226-2. Epub 2021 Mar 4. Pharmacol Rep. 2021. PMID: 33661518
In this work, we describe the development of novel 4-phenylbutyric acid (4-PBA) lysosome/ER-targeted derivatives. We assumed that 4-PBA targeting to specific organelles, where protein degradation takes place, might reduce the 4-PBA effective concentration. ...
In this work, we describe the development of novel 4-phenylbutyric acid (4-PBA) lysosome/ER-targeted derivatives. We assumed t …
Inhibition of Renal Tubular Epithelial Mesenchymal Transition and Endoplasmic Reticulum Stress-Induced Apoptosis with Shenkang Injection Attenuates Diabetic Tubulopathy.
Wang WW, Liu YL, Wang MZ, Li H, Liu BH, Tu Y, Yuan CC, Fang QJ, Chen JX, Wang J, Fu Y, Wan ZY, Wan YG, Wu W. Wang WW, et al. Front Pharmacol. 2021 Aug 2;12:662706. doi: 10.3389/fphar.2021.662706. eCollection 2021. Front Pharmacol. 2021. PMID: 34408650 Free PMC article.
For in vitro research, we exposed NRK-52E cells to high glucose (HG) and 4-phenylbutyric acid (4-PBA) with or without SKI or ROS. Changes in parameters related to renal tubular injury and EMT were analyzed in vivo. ...
For in vitro research, we exposed NRK-52E cells to high glucose (HG) and 4-phenylbutyric acid (4-PBA) with or without SKI or R …
The host protein SSR4 mediates PRRSV-induced endoplasmic reticulum stress via interaction with Nsp2.
Li Y, Gao H, Liu Z, Lu M, Shen Y, Xing Y, Wang Y, Wu X, Yang P, Yuan H, Hou Y, Cai Y, Li B, Xiao Y. Li Y, et al. J Virol. 2026 May 19;100(5):e0026626. doi: 10.1128/jvi.00266-26. Epub 2026 Apr 23. J Virol. 2026. PMID: 42023815 Free PMC article.
This relationship is finely balanced, as pharmacological disruption of ER homeostasis using either the inducer tunicamycin (TU) or the chemical chaperone 4-phenylbutyric acid (4-PBA) potently inhibited viral replication. Importantly, TU and another ind …
This relationship is finely balanced, as pharmacological disruption of ER homeostasis using either the inducer tunicamycin (TU) or th …