Tuning protein folding in lysosomal storage diseases: the chemistry behind pharmacological chaperones
Chemical Science Pub Date: 2018-01-10 DOI: 10.1039/C7SC04712F
Abstract
Misfolding of proteins is the basis of several proteinopathies. Chemical and pharmacological chaperones are small molecules capable of inducing the correct conformation of proteins, thus being of interest for human therapeutics. The most recent developments in medicinal chemistry and in the drug development of pharmacological chaperones are discussed, with focus on lysosomal storage diseases.
Recommended Literature
- [1] Establishment and implications of a characterization method for magnetic nanoparticle using cell tracking velocimetry and magnetic susceptibility modified solutions Huading Zhang,Lee R. Moore,Maciej Zborowski,P. Stephen Williams,Shlomo Margel,Jeffrey J. ChalmersAnalyst, 2005,130, 514-527 10.1039/B412723D
- [2] Evolution of hierarchical porous structures in supramolecular guest–host hydrogels? Christopher B. Rodell,Christopher B. Highley,Minna H. Chen,Neville N. Dusaj,Chao Wang,Lin Han,Jason A. BurdickSoft Matter, 2016,12, 7839-7847 10.1039/C6SM01395C
- [3] Excimer emission and magnetoluminescence of radical-based zinc(ii) complexes doped in host crystals? Shojiro Kimura,Tetsuro KusamotoChem. Commun., 2020,56, 11195-11198 10.1039/D0CC04830E
- [4] Exchanged ligands on the surface of a giant cluster: [(MoO3)176(H2O)63(CH3OH)17Hn](32 – n)– Chem. Commun., 1998, 1501-1502 10.1039/A801804I
- [5] Excitonic and vibrational coherence in artificial photosynthetic systems studied by negative-time ultrafast laser spectroscopy Dongjia Han,Bing Xue,Juan Du,Tomohiro Miyatake,Hitoshi Tamiaki,Xin Xing,Wei Yuan,Yanyan LiPhys. Chem. Chem. Phys., 2016,18, 24252-24260 10.1039/C6CP03540J
- [6] Dissolution of cork biopolymers in biocompatible ionic liquids Helga Garcia,Rui Ferreira,Marija Petkovic,Jamie L. Ferguson,Maria C. Leit?o,H. Q. Nimal Gunaratne,Luís Paulo N. RebeloGreen Chem., 2010,12, 367-369 10.1039/B922553F
- [7] Fast synthesis of copper nanoclusters through the use of hydrogen peroxide additive and their application for the fluorescence detection of Hg2+ in water samples? Liao Xiaoqing,Li Ruiyi,Li Zaijun,Sun Xiulan,Wang Zhouping,Liu JunkangNew J. Chem., 2015,39, 5240-5248 10.1039/C5NJ00831J
- [8] Evolution of dealloying induced strain in nanoporous gold crystals? Ross Harder,David C. Dunand,Ian McNultyNanoscale, 2017,9, 5686-5693 10.1039/C6NR09635B
- [9] Distinct impact of glycation towards the aggregation and toxicity of murine and human amyloid-β? Eunju Nam,Jiyeon Han,Sunhee Choi,Mi Hee LimChem. Commun., 2021,57, 7637-7640 10.1039/D1CC02695J
- [10] Evolution in surface coverage of CH3NH3PbI3?XClXvia heat assisted solvent vapour treatment and their effects on photovoltaic performance of devices Dhirendra K. Chaudhary,Pramendra Kumar,Lokendra KumarRSC Adv., 2016,6, 94731-94738 10.1039/C6RA18729C
Journal Name：Chemical Science
research_products
-
CAS no.: 89640-58-4
![1,2,3,4,5,6-Hexahydro-[2,3]bipyridinyl-6-ol](https://ossimg.kuujia.com/scimg/1271000/1270529-38-8x500.png)
![N-(3-cyanothiolan-3-yl)-2-[(2,2,2-trifluoroethyl)sulfanyl]pyridine-4-carboxamide](https://ossimg.kuujia.com/scimg/1444500/1444113-98-7x500.png)
![2,2-Difluoro-3-[methyl(2-methylbutyl)amino]propanoic acid](https://ossimg.kuujia.com/scimg/2138500/2138166-62-6x500.png)
![2-(1H-indol-3-yl)-N-{[6-(thiophen-2-yl)-[1,2,4]triazolo[4,3-b]pyridazin-3-yl]methyl}acetamide](https://ossimg.kuujia.com/scimg/2034500/2034271-14-0x500.png)