Giordani Antonio, Menziani Maria Cristina, Moresco Rosa Maria, Matarrese Mario, Paolino Marco, Saletti Mario, Giuliani Germano, Anzini Maurizio, Cappelli Andrea
Rottapharm Biotech S.p.A., Via Valosa di Sopra 9, 20900 Monza, Italy.
Dipartimento di Scienze Chimiche e Geologiche, Università di Modena e Reggio Emilia, Via Campi 103, 41121 Modena, Italy.
J Med Chem. 2021 Jul 22;64(14):9649-9676. doi: 10.1021/acs.jmedchem.1c00379. Epub 2021 Jul 13.
Translocator protein 18 kDa [TSPO or peripheral-type benzodiazepine receptor (PBR)] was identified in the search of binding sites for benzodiazepine anxiolytic drugs in peripheral regions. In these areas, binding sites for TSPO ligands were recognized in steroid-producing tissues. TSPO plays an important role in many cellular functions, and its coding sequence is highly conserved across species. TSPO is located predominantly on the membrane of mitochondria and is overexpressed in several solid cancers. TSPO basal expression in the CNS is low, but it becomes high in neurodegenerative conditions. Thus, TSPO constitutes not only as an outstanding drug target but also as a valuable marker for the diagnosis of a number of diseases. The aim of the present article is to show the lesson we have learned from our activity in TSPO medicinal chemistry and in approaching the targeted delivery to mitochondria by means of TSPO ligands.
18 kDa转位蛋白[TSPO或外周型苯二氮䓬受体(PBR)]是在寻找外周区域苯二氮䓬抗焦虑药物结合位点的过程中发现的。在这些区域,TSPO配体的结合位点在类固醇生成组织中被识别。TSPO在许多细胞功能中发挥重要作用,其编码序列在物种间高度保守。TSPO主要位于线粒体外膜,在几种实体癌中过表达。TSPO在中枢神经系统中的基础表达较低,但在神经退行性疾病中会升高。因此,TSPO不仅是一个出色的药物靶点,也是多种疾病诊断的有价值标志物。本文的目的是展示我们在TSPO药物化学以及通过TSPO配体实现线粒体靶向递送方面所学到的经验。