Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX 77030, USA; Jan and Dan Duncan Neurological Research Institute, Texas Children's Hospital, Houston, TX 77030, USA; Therapeutic Innovation Center (THINC), Baylor College of Medicine, Houston, TX 77030, USA; Center for Alzheimer's and Neurodegenerative Diseases (CAND), Texas Children's Hospital, Houston, TX 77030, USA; Dan L Duncan Comprehensive Cancer Center (DLDCCC), Baylor College of Medicine, Houston, TX 77030, USA; Department of Genetics, Stanford University, Stanford, CA 94305, USA.
Department of Plant Biology, Carnegie Institution for Science, Stanford, CA 94305, USA; Department of Biology, Stanford University, Stanford, CA 94305, USA.
Mol Cell. 2023 Jun 15;83(12):2020-2034.e6. doi: 10.1016/j.molcel.2023.05.025. Epub 2023 Jun 8.
Biomolecular condensation underlies the biogenesis of an expanding array of membraneless assemblies, including stress granules (SGs), which form under a variety of cellular stresses. Advances have been made in understanding the molecular grammar of a few scaffold proteins that make up these phases, but how the partitioning of hundreds of SG proteins is regulated remains largely unresolved. While investigating the rules that govern the condensation of ataxin-2, an SG protein implicated in neurodegenerative disease, we unexpectedly identified a short 14 aa sequence that acts as a condensation switch and is conserved across the eukaryote lineage. We identify poly(A)-binding proteins as unconventional RNA-dependent chaperones that control this regulatory switch. Our results uncover a hierarchy of cis and trans interactions that fine-tune ataxin-2 condensation and reveal an unexpected molecular function for ancient poly(A)-binding proteins as regulators of biomolecular condensate proteins. These findings may inspire approaches to therapeutically target aberrant phases in disease.
生物分子凝聚是一系列无膜组装体生物发生的基础,包括应激颗粒(SGs),它们在多种细胞应激下形成。在理解构成这些相的少数支架蛋白的分子语法方面已经取得了进展,但如何调节数百种 SG 蛋白的分区仍然在很大程度上没有得到解决。在研究参与神经退行性疾病的 SG 蛋白 ataxin-2 凝聚规则时,我们意外地发现了一个短的 14 个氨基酸序列,它作为凝聚开关,在真核生物谱系中保守。我们确定 poly(A)-结合蛋白是非常规的 RNA 依赖性伴侣蛋白,它们控制这个调节开关。我们的结果揭示了精细调节 ataxin-2 凝聚的顺式和反式相互作用的层次结构,并揭示了古老的 poly(A)-结合蛋白作为生物分子凝聚蛋白调节剂的意外分子功能。这些发现可能为治疗性靶向疾病中异常相提供了方法。