Adli Mazhar, Xing De, Bai Tao, Neyisci Ozlem, Paylakhi Seyedehzahra, Duval Alexander, Tekin Yasemin
Northwestern University, Feinberg School of Medicine.
Res Sq. 2024 Nov 15:rs.3.rs-5348956. doi: 10.21203/rs.3.rs-5348956/v1.
Biological mechanisms are inherently dynamic, requiring precise and rapid gene manipulation for effective characterization. Traditional genetic perturbation tools such as siRNA and CRISPR knockout operate on timescales that render them unsuitable for exploring dynamic processes or studying essential genes, where chronic depletion can lead to cell death. Here, we compared four major inducible degron systems-dTAG, HaloPROTAC, and two auxin-inducible degron (AID) tools-in human pluripotent stem cells. We evaluated basal degradation levels, inducible degradation kinetics, and recovery dynamics for endogenously tagged genes. While the AID 2.0 system is the most efficient for rapid protein degradation, it exhibited higher basal degradation and slower recovery after ligand washout. To address these challenges, we applied directed protein evolution, incorporating base-editing-mediated mutagenesis and iterative functional selection and screening. We discovered novel OsTIR1 variants, including S210A, with significantly enhanced overall degron efficiency. The resulting system, designated as AID 3.0, demonstrates minimal basal degradation and rapid and effective target protein depletion and substantially rescues the cellular and molecular phenotypes due to basal degradation or slow target protein recovery in previous systems. We conclude that AID 3.0 represents a superior degron technology, offering a valuable tool for studying gene functions in dynamic biological contexts and exploring therapeutic applications. Additionally, the research strategy used here could be broadly applicable for improving other degron and biological tools.
生物机制本质上是动态的,需要精确且快速的基因操作才能进行有效的表征。传统的基因扰动工具,如小干扰RNA(siRNA)和CRISPR基因敲除,其作用时间尺度使其不适用于探索动态过程或研究必需基因,因为长期缺失可能导致细胞死亡。在此,我们在人类多能干细胞中比较了四种主要的诱导性降解子系统——dTAG、HaloPROTAC和两种生长素诱导性降解子(AID)工具。我们评估了内源性标记基因的基础降解水平、诱导性降解动力学和恢复动力学。虽然AID 2.0系统在快速蛋白质降解方面效率最高,但它表现出更高的基础降解水平,并且在去除配体后恢复较慢。为应对这些挑战,我们应用了定向蛋白质进化,结合碱基编辑介导的诱变以及迭代功能选择和筛选。我们发现了新型的OsTIR1变体,包括S210A,其整体降解子效率显著提高。由此产生的系统,命名为AID 3.0,表现出最小的基础降解以及快速有效的靶蛋白去除,并且极大地挽救了先前系统中由于基础降解或靶蛋白恢复缓慢而导致的细胞和分子表型。我们得出结论,AID 3.0代表了一种更优越的降解子技术,为在动态生物学背景下研究基因功能和探索治疗应用提供了一种有价值的工具。此外,这里使用的研究策略可广泛应用于改进其他降解子和生物学工具。