{"title":"[通过PROTACs诱导蛋白质降解和其他策略:走向有前途的药物]。","authors":"Michèle Reboud-Ravaux","doi":"10.1051/jbio/2021007","DOIUrl":null,"url":null,"abstract":"<p><p>Targeted protein degradation (TPD), discovered twenty years ago through the PROTAC technology, is rapidly developing thanks to the implication of many scientists from industry and academia. PROTAC chimeras are heterobifunctional molecules able to link simultaneously a protein to be degraded and an E3 ubiquitin ligase. This allows the protein ubiquitination and its degradation by 26S proteasome. PROTACs have evolved from small peptide molecules to small non-peptide and orally available molecules. It was shown that PROTACs are capable to degrade proteins considered as \"undruggable\" i.e. devoid of well-defined pockets and deep grooves possibly occupied by small molecules. Among these \"hard to drug\" proteins, several can be degraded by PROTACs: scaffold proteins, BAF complex, transcription factors, Ras family proteins. Two PROTACs are clinically tested for breast (ARV471) and prostate (ARV110) cancers. The protein degradation by proteasome is also induced by other types of molecules: molecular glues, hydrophobic tagging (HyT), HaloPROTACs and homo-PROTACs. Other cellular constituents are eligible to induced degradation: RNA-PROTACs for RNA binding proteins and RIBOTACs for degradation of RNA itself (SARS-CoV-2 RNA). TPD has recently moved beyond the proteasome with LYTACs (lysosome targeting chimeras) and MADTACs (macroautophagy degradation targeting chimeras). Several techniques such as screening platforms together with mathematical modeling and computational design are now used to improve the discovery of new efficient PROTACs.</p>","PeriodicalId":39068,"journal":{"name":"Biologie Aujourd''hui","volume":"215 1-2","pages":"25-43"},"PeriodicalIF":0.0000,"publicationDate":"2021-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"1","resultStr":"{\"title\":\"[Induced degradation of proteins by PROTACs and other strategies: towards promising drugs].\",\"authors\":\"Michèle Reboud-Ravaux\",\"doi\":\"10.1051/jbio/2021007\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>Targeted protein degradation (TPD), discovered twenty years ago through the PROTAC technology, is rapidly developing thanks to the implication of many scientists from industry and academia. PROTAC chimeras are heterobifunctional molecules able to link simultaneously a protein to be degraded and an E3 ubiquitin ligase. This allows the protein ubiquitination and its degradation by 26S proteasome. PROTACs have evolved from small peptide molecules to small non-peptide and orally available molecules. It was shown that PROTACs are capable to degrade proteins considered as \\\"undruggable\\\" i.e. devoid of well-defined pockets and deep grooves possibly occupied by small molecules. Among these \\\"hard to drug\\\" proteins, several can be degraded by PROTACs: scaffold proteins, BAF complex, transcription factors, Ras family proteins. Two PROTACs are clinically tested for breast (ARV471) and prostate (ARV110) cancers. The protein degradation by proteasome is also induced by other types of molecules: molecular glues, hydrophobic tagging (HyT), HaloPROTACs and homo-PROTACs. Other cellular constituents are eligible to induced degradation: RNA-PROTACs for RNA binding proteins and RIBOTACs for degradation of RNA itself (SARS-CoV-2 RNA). TPD has recently moved beyond the proteasome with LYTACs (lysosome targeting chimeras) and MADTACs (macroautophagy degradation targeting chimeras). Several techniques such as screening platforms together with mathematical modeling and computational design are now used to improve the discovery of new efficient PROTACs.</p>\",\"PeriodicalId\":39068,\"journal\":{\"name\":\"Biologie Aujourd''hui\",\"volume\":\"215 1-2\",\"pages\":\"25-43\"},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2021-01-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"1\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Biologie Aujourd''hui\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1051/jbio/2021007\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2021/8/16 0:00:00\",\"PubModel\":\"Epub\",\"JCR\":\"Q4\",\"JCRName\":\"Biochemistry, Genetics and Molecular Biology\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Biologie Aujourd''hui","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1051/jbio/2021007","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2021/8/16 0:00:00","PubModel":"Epub","JCR":"Q4","JCRName":"Biochemistry, Genetics and Molecular Biology","Score":null,"Total":0}
引用次数: 1
摘要
20年前通过PROTAC技术发现的靶向蛋白降解(Targeted protein degradation, TPD),在工业界和学术界许多科学家的推动下得到了迅速发展。PROTAC嵌合体是一种异功能分子,能够同时连接待降解蛋白和E3泛素连接酶。这允许蛋白质泛素化和26S蛋白酶体降解。protac已经从小肽分子进化到小的非肽分子和口服分子。研究表明,PROTACs能够降解被认为是“不可药物”的蛋白质,即没有明确定义的口袋和可能被小分子占据的深沟槽。在这些“难以药物化”的蛋白中,有几种可以被PROTACs降解:支架蛋白、BAF复合物、转录因子、Ras家族蛋白。两种PROTACs用于乳腺癌(ARV471)和前列腺癌(ARV110)的临床试验。蛋白酶体的蛋白质降解也可由其他类型的分子诱导:分子胶、疏水标记(HyT)、HaloPROTACs和homo-PROTACs。其他细胞成分也适合诱导降解:RNA结合蛋白的RNA- protacs和RNA本身降解的RIBOTACs (SARS-CoV-2 RNA)。最近,TPD已经超越了蛋白酶体,有LYTACs(靶向嵌合体的溶酶体)和MADTACs(靶向嵌合体的巨噬降解)。筛选平台、数学建模和计算设计等几种技术现在被用于改进新型高效protac的发现。
[Induced degradation of proteins by PROTACs and other strategies: towards promising drugs].
Targeted protein degradation (TPD), discovered twenty years ago through the PROTAC technology, is rapidly developing thanks to the implication of many scientists from industry and academia. PROTAC chimeras are heterobifunctional molecules able to link simultaneously a protein to be degraded and an E3 ubiquitin ligase. This allows the protein ubiquitination and its degradation by 26S proteasome. PROTACs have evolved from small peptide molecules to small non-peptide and orally available molecules. It was shown that PROTACs are capable to degrade proteins considered as "undruggable" i.e. devoid of well-defined pockets and deep grooves possibly occupied by small molecules. Among these "hard to drug" proteins, several can be degraded by PROTACs: scaffold proteins, BAF complex, transcription factors, Ras family proteins. Two PROTACs are clinically tested for breast (ARV471) and prostate (ARV110) cancers. The protein degradation by proteasome is also induced by other types of molecules: molecular glues, hydrophobic tagging (HyT), HaloPROTACs and homo-PROTACs. Other cellular constituents are eligible to induced degradation: RNA-PROTACs for RNA binding proteins and RIBOTACs for degradation of RNA itself (SARS-CoV-2 RNA). TPD has recently moved beyond the proteasome with LYTACs (lysosome targeting chimeras) and MADTACs (macroautophagy degradation targeting chimeras). Several techniques such as screening platforms together with mathematical modeling and computational design are now used to improve the discovery of new efficient PROTACs.