Battaglia Laura, Scorrano Giovanna, Spiaggia Rossana, Basile Antonio, Palmucci Stefano, Foti Pietro Valerio, Spatola Corrado, Iacomino Michele, Marinangeli Franco, Francia Elisa, Comisi Francesco, Corsello Antonio, Salpietro Vincenzo, Vittori Alessandro, David Emanuele
Department of Medical Surgical Sciences and Advanced Technologies "GF Ingrassia", University Hospital Policlinic "G. Rodolico-San Marco", Catania, Italy.
Department of Biotechnological and Applied Clinical Sciences, University of L'Aquila, L'Aquila, Italy.
Front Pediatr. 2023 Dec 15;11:1301166. doi: 10.3389/fped.2023.1301166. eCollection 2023.
The WWOX gene encodes a 414-amino-acid protein composed of two N-terminal WW domains and a C-terminal short-chain dehydrogenase/reductase (SDR) domain. WWOX protein is highly conserved among species and mainly expressed in the cerebellum, cerebral cortex, brain stem, thyroid, hypophysis, and reproductive organs. It plays a crucial role in the biology of the central nervous system, and it is involved in neuronal development, migration, and proliferation. Biallelic pathogenic variants in WWOX have been associated with an early infantile epileptic encephalopathy known as WOREE syndrome. Both missense and null variants have been described in affected patients, leading to a reduction in protein function and stability. The most severe WOREE phenotypes have been related to biallelic null/null variants, associated with the complete loss of function of the protein. All affected patients showed brain anomalies on magnetic resonance imaging (MRI), suggesting the pivotal role of WWOX protein in brain homeostasis and developmental processes. We provided a literature review, exploring both the clinical and radiological spectrum related to WWOX pathogenic variants, described to date. We focused on neuroradiological findings to better delineate the WOREE phenotype with diagnostic and prognostic implications.
WWOX基因编码一种由两个N端WW结构域和一个C端短链脱氢酶/还原酶(SDR)结构域组成的414个氨基酸的蛋白质。WWOX蛋白在物种间高度保守,主要在小脑、大脑皮层、脑干、甲状腺、垂体和生殖器官中表达。它在中枢神经系统生物学中起关键作用,参与神经元发育、迁移和增殖。WWOX的双等位基因致病性变异与一种称为WOREE综合征的早发性婴儿癫痫性脑病相关。在受影响的患者中已描述了错义变异和无效变异,导致蛋白质功能和稳定性降低。最严重的WOREE表型与双等位基因无效/无效变异有关,与蛋白质功能的完全丧失相关。所有受影响的患者在磁共振成像(MRI)上均显示脑异常,提示WWOX蛋白在脑内稳态和发育过程中的关键作用。我们提供了一篇文献综述,探讨了迄今为止所描述的与WWOX致病性变异相关的临床和放射学谱。我们专注于神经放射学发现,以更好地描绘具有诊断和预后意义的WOREE表型。