Division of Hematology/Oncology, Boston Children's Hospital, Department of Pediatric Oncology, Dana-Farber Cancer Institute, Harvard Stem Cell Institute, Broad Institute, Department of Pediatrics, Harvard Medical School, Boston, MA, USA.
Molecular Pathology Unit, Center for Cancer Research, Massachusetts General Hospital, Department of Pathology, Harvard Medical School, Boston, MA, USA.
Nat Genet. 2021 May;53(5):719-728. doi: 10.1038/s41588-021-00843-w. Epub 2021 Apr 15.
Known fetal hemoglobin (HbF) silencers have potential on-target liabilities for rational β-hemoglobinopathy therapeutic inhibition. Here, through transcription factor (TF) CRISPR screening, we identify zinc-finger protein (ZNF) 410 as an HbF repressor. ZNF410 does not bind directly to the genes encoding γ-globins, but rather its chromatin occupancy is concentrated solely at CHD4, encoding the NuRD nucleosome remodeler, which is itself required for HbF repression. CHD4 has two ZNF410-bound regulatory elements with 27 combined ZNF410 binding motifs constituting unparalleled genomic clusters. These elements completely account for the effects of ZNF410 on fetal globin repression. Knockout of ZNF410 or its mouse homolog Zfp410 reduces CHD4 levels by 60%, enough to substantially de-repress HbF while eluding cellular or organismal toxicity. These studies suggest a potential target for HbF induction for β-hemoglobin disorders with a wide therapeutic index. More broadly, ZNF410 represents a special class of gene regulator, a conserved TF with singular devotion to regulation of a chromatin subcomplex.
已知的胎儿血红蛋白 (HbF) 沉默子在合理的 β-血红蛋白病治疗抑制方面具有潜在的靶点缺陷。在这里,我们通过转录因子 (TF) CRISPR 筛选,鉴定锌指蛋白 (ZNF) 410 为 HbF 抑制剂。ZNF410 并不直接与编码 γ-球蛋白的基因结合,而是其染色质占有率仅集中在 CHD4 上,CHD4 编码 NuRD 核小体重塑酶,该酶本身是 HbF 抑制所必需的。CHD4 有两个 ZNF410 结合的调节元件,有 27 个结合的 ZNF410 结合基序,构成了无与伦比的基因组簇。这些元件完全解释了 ZNF410 对胎儿球蛋白抑制的影响。ZNF410 或其小鼠同源物 Zfp410 的敲除使 CHD4 水平降低 60%,足以大大解除 HbF 的抑制作用,同时避免细胞或机体毒性。这些研究为具有广泛治疗指数的 β-血红蛋白疾病的 HbF 诱导提供了一个潜在的靶点。更广泛地说,ZNF410 代表了一类特殊的基因调节剂,是一种保守的 TF,专门致力于调节染色质亚复合物。