Leclair Nathan K, Choudury Abrar, Chen William C, Magill Stephen T, McCortney Kathleen, Horbinski Craig M, Chen Zhenhong, Goldschmidt Ezequiel, Eaton Charlotte D, Bulsara Ketan R, Bi Wenya Linda, Patel Akash J, Sahm Felix, Raleigh David, Anczukow Olga
Graduate Program in Genetics and Development, UConn Health, Farmington, CT, USA.
The Jackson Laboratory for Genomic Medicine, Farmington, CT, USA.
Neuro Oncol. 2024 Dec 5;26(12):2222-2236. doi: 10.1093/neuonc/noae150.
Advances in our understanding of the molecular biology of meningiomas have led to significant gains in the ability to predict patient prognosis and tumor recurrence and to identify novel targets for therapeutic design. Specifically, classification of meningiomas based on DNA methylation has greatly improved our ability to risk stratify patients, however new questions have arisen in terms of the underlying impact these DNA-methylation signatures have on meningioma biology.
This study utilizes RNA-sequencing data from 486 meningioma samples corresponding to 3 meningioma DNA-methylation groups (merlin-intact, immune-enriched, and hypermitotic), followed by in vitro experiments utilizing human meningioma cell lines.
We identify alterations in RNA splicing between meningioma DNA-methylation groups including individual splicing events that correlate with hypermitotic meningiomas and predict tumor recurrence and overall patient prognosis and compile a set of splicing events that can accurately predict DNA-methylation classification based on RNA-seq data. Furthermore, we validate these events using reverse transcription polymerase chain reaction (RT-PCR) in patient samples and meningioma cell lines. Additionally, we identify alterations in RNA-binding proteins and splicing factors that lie upstream of RNA splicing events, including upregulation of SRSF1 in hypermitotic meningiomas which we show drives alternative RNA splicing changes. Finally, we design splice-switching antisense oligonucleotides to target RNA splicing changes in NASP and MFF observed in hypermitotic meningiomas, providing a rationale for RNA-based therapeutic design.
RNA splicing is an important driver of meningioma phenotypes that can be useful in prognosticating patients and as a potential exploit for therapeutic vulnerabilities.
我们对脑膜瘤分子生物学认识的进展,已使预测患者预后和肿瘤复发的能力以及识别治疗设计新靶点的能力有了显著提高。具体而言,基于DNA甲基化的脑膜瘤分类极大地提升了我们对患者进行风险分层的能力,然而,这些DNA甲基化特征对脑膜瘤生物学的潜在影响引发了新的问题。
本研究利用了来自486个脑膜瘤样本的RNA测序数据,这些样本对应3种脑膜瘤DNA甲基化组(merlin完整组、免疫富集组和高有丝分裂组),随后利用人脑膜瘤细胞系进行了体外实验。
我们识别出脑膜瘤DNA甲基化组之间RNA剪接的改变,包括与高有丝分裂性脑膜瘤相关且能预测肿瘤复发和患者总体预后的个别剪接事件,并编制了一组能基于RNA测序数据准确预测DNA甲基化分类的剪接事件。此外,我们在患者样本和脑膜瘤细胞系中使用逆转录聚合酶链反应(RT-PCR)验证了这些事件。另外,我们识别出RNA剪接事件上游的RNA结合蛋白和剪接因子的改变,包括高有丝分裂性脑膜瘤中SRSF1的上调,我们证明其驱动了替代性RNA剪接变化。最后,我们设计了剪接转换反义寡核苷酸,以靶向高有丝分裂性脑膜瘤中观察到的NASP和MFF的RNA剪接变化,为基于RNA的治疗设计提供了理论依据。
RNA剪接是脑膜瘤表型的重要驱动因素,可用于患者预后评估,并作为治疗靶点的潜在开发方向。