先驱转录因子PU.1在急性髓系白血病（AML）关键增强子的形成中起早期核心作用，与SWI/SNF协同推动MYC等关键致癌基因的表达。通过抑制SWI/SNF或干扰PU.1与DNA的结合，可有效破坏AML中的增强子结构，导致肿瘤细胞分化与增殖抑制。靶向PU.1-SWI/SNF调控轴为肿瘤精准治疗提供了新的方向。结合CUT&Tag与点击化学（Click Chemistry）开发的“CLICK-on-CUT&Tag”技术为精准识别PU.1功能靶点并诱导转录因子重新分布提供了有力工具。
Why does the same gene cause a phenotypic change when knocked down but not when knocked out? The disconnection between genotype and expected phenotype in such cases may be attributed to the activation of the Genetic Compensation Response (GCR). When a gene is mutated or knocked out, GCR is triggered, compensating for the lost function by upregulating homologous or functionally similar genes. This response is particularly pronounced when the mRNA contains a premature termination codon (PTC). GCR is not only a protective mechanism ensuring genetic robustness, but it may also play a significant role in influencing cancer progression. Understanding the mechanisms behind GCR is crucial for advancing our knowledge of tumor progression and for identifying new therapeutic targets.