Pasqualetto, Gaia published the artcileLigand-based rational design, synthesis and evaluation of novel potential chemical chaperones for opsin, Recommanded Product: tert-Butyl piperidin-4-ylcarbamate, the main research area is rhodopsin binding site chromophore preparation chem chaperone opsin; cyclohexene preparation rhodopsin binding site chromophore chem chaperone opsin; Molecular modelling; Rhodopsin; Severe inherited blinding diseases; Small-molecule agents; Synthetic organic chemistry.
Inherited blinding diseases retinitis pigmentosa (RP) and a subset of Leber’s congenital amaurosis (LCA) are caused by the misfolding and mistrafficking of rhodopsin mols., which aggregate and accumulate in the endoplasmic reticulum (ER), leading to photoreceptor cell death. One potential therapeutic strategy to prevent the loss of photoreceptors in these conditions is to identify opsin-binding compounds that act as chem. chaperones for opsin, aiding its proper folding and trafficking to the outer cell membrane. Aiming to identify novel compounds with such effect, a rational ligand-based approach was applied to the structure of the visual pigment chromophore, 11-cis-retinal, and its locked analog 11-cis-6mr-retinal. Following mol. docking studies on the main chromophore binding site of rhodopsin, 49 novel compounds, e.g., I, were synthesized according to optimized one-to seven-step synthetic routes. These agents were evaluated for their ability to compete for the chromophore binding site of opsin, and their capacity to increase the trafficking of the P23H opsin mutant from the ER to the cell membrane. Different new mols. displayed an effect in at least one assay, acting either as chem. chaperones or as stabilizers of the 9-cis-retinal-rhodopsin complex. These compounds could provide the basis to develop novel therapeutics for RP and LCA.
European Journal of Medicinal Chemistry published new progress about Cytotoxicity. 73874-95-0 belongs to class piperidines, name is tert-Butyl piperidin-4-ylcarbamate, and the molecular formula is C10H20N2O2, Recommanded Product: tert-Butyl piperidin-4-ylcarbamate.
Referemce:
Piperidine – Wikipedia,
Piperidine | C5H11N – PubChem