School of Biomedical Sciences, Li Ka Shing Faculty of Medicine, The University of Hong Kong, Hong Kong SAR, China.
Centre for Translational Stem Cell Biology, Hong Kong.
Nucleic Acids Res. 2023 Sep 22;51(17):8934-8956. doi: 10.1093/nar/gkad597.
An engineered SOX17 variant with point mutations within its DNA binding domain termed SOX17FNV is a more potent pluripotency inducer than SOX2, yet the underlying mechanism remains unclear. Although wild-type SOX17 was incapable of inducing pluripotency, SOX17FNV outperformed SOX2 in mouse and human pluripotency reprogramming. In embryonic stem cells, SOX17FNV could replace SOX2 to maintain pluripotency despite considerable sequence differences and upregulated genes expressed in cleavage-stage embryos. Mechanistically, SOX17FNV co-bound OCT4 more cooperatively than SOX2 in the context of the canonical SoxOct DNA element. SOX2, SOX17, and SOX17FNV were all able to bind nucleosome core particles in vitro, which is a prerequisite for pioneer transcription factors. Experiments using purified proteins and in cellular contexts showed that SOX17 variants phase-separated more efficiently than SOX2, suggesting an enhanced ability to self-organise. Systematic deletion analyses showed that the N-terminus of SOX17FNV was dispensable for its reprogramming activity. However, the C-terminus encodes essential domains indicating multivalent interactions that drive transactivation and reprogramming. We defined a minimal SOX17FNV (miniSOX) that can support reprogramming with high activity, reducing the payload of reprogramming cassettes. This study uncovers the mechanisms behind SOX17FNV-induced pluripotency and establishes engineered SOX factors as powerful cell engineering tools.
一种具有 DNA 结合域点突变的工程化 SOX17 变体,称为 SOX17FNV,比 SOX2 更能有效地诱导多能性,但潜在的机制尚不清楚。虽然野生型 SOX17 不能诱导多能性,但 SOX17FNV 在小鼠和人类多能性重编程中比 SOX2 更有效。在胚胎干细胞中,尽管 SOX17FNV 与 SOX2 存在相当大的序列差异和上调的基因在卵裂期胚胎中表达,但 SOX17FNV 可以替代 SOX2 来维持多能性。从机制上讲,在典型的 SoxOct DNA 元件中,SOX17FNV 比 SOX2 更能协同地与 OCT4 结合。SOX2、SOX17 和 SOX17FNV 都能够在体外与核小体核心颗粒结合,这是先驱转录因子的先决条件。使用纯化蛋白和细胞环境进行的实验表明,SOX17 变体比 SOX2 更有效地相分离,这表明其自我组织的能力增强。系统的删除分析表明,SOX17FNV 的 N 端对于其重编程活性是可有可无的。然而,C 端编码必需的结构域,表明多价相互作用驱动转录激活和重编程。我们定义了一个最小的 SOX17FNV(miniSOX),它可以以高活性支持重编程,减少重编程盒的有效载荷。这项研究揭示了 SOX17FNV 诱导多能性的背后机制,并确立了工程化 SOX 因子作为强大的细胞工程工具。