Department of Bioengineering, University of California, Berkeley, Berkeley, CA 94720, USA.
Department of Chemistry, University of California, Berkeley, Berkeley, CA 94720, USA.
Curr Opin Biotechnol. 2018 Aug;52:25-31. doi: 10.1016/j.copbio.2018.02.009. Epub 2018 Feb 24.
Protein therapeutics based on transcription factors, gene editing enzymes, signaling proteins and protein antigens, have the potential to provide cures for a wide number of untreatable diseases, but cannot be developed into therapeutics due to challenges in delivering them into the cytoplasm. There is therefore great interest in developing strategies that can enable proteins to enter the cytoplasm of cells. In this review article we will discuss recent progress in intracellular protein therapeutics, which are focused on the following four classes of therapeutics, Firstly, vaccine development, secondly, transcription factor therapies, thirdly, gene editing and finally, cancer therapeutics. These exciting new advances raise the prospect of developing cures for several un-treatable diseases.
基于转录因子、基因编辑酶、信号蛋白和蛋白抗原的蛋白质疗法有可能为许多无法治疗的疾病提供治愈方法,但由于将它们递送到细胞质中存在挑战,因此无法开发成治疗方法。因此,人们非常有兴趣开发能够使蛋白质进入细胞细胞质的策略。在这篇综述文章中,我们将讨论细胞内蛋白质治疗的最新进展,重点介绍以下四类治疗方法:首先,疫苗开发;其次,转录因子治疗;第三,基因编辑;最后,癌症治疗。这些令人兴奋的新进展提出了为几种无法治疗的疾病开发治疗方法的前景。