Vitorino Marta, Cunha Nídia, Conceição Natércia, Cancela M Leonor
Center of Marine Sciences (CCMAR), University of Algarve, 8005-139, Faro, Portugal.
Department of Biomedical Sciences and Medicine, University of Algarve, 8005-139, Faro, Portugal.
Mol Biol Rep. 2018 Aug;45(4):445-451. doi: 10.1007/s11033-018-4180-1. Epub 2018 May 11.
Atypical Rett syndrome is a child neurodevelopmental disorder induced by mutations in CDKL5 gene and characterized by a progressive regression in development with loss of purposeful use of the hands, slowed brain and head growth, problems with walking, seizures, and intellectual disability. At the moment, there is no cure for this pathology and little information is available concerning animal models capable of mimicking its phenotypes, thus the development of additional animal models should be of interest to gain more knowledge about the disease. Zebrafish has been used successfully as model organism for many human genetic diseases; however, no information is available concerning the spatial and temporal expression of cdkl5 orthologous in this organism. In the present study, we identified the developmental expression patterns of cdkl5 in zebrafish by quantitative PCR and whole-mount in situ hybridization. cdkl5 is expressed maternally at low levels during the first 24 h of development. After that the expression of the gene increases significantly and it starts to be expressed mainly in the nervous system and in several brain structures, such as telencephalon, mesencephalon and diencephalon. The expression patterns of cdkl5 in zebrafish is in accordance with the tissues known to be affected in humans and associated to symptoms and deficits observed in Rett syndrome patients thus providing the first evidence that zebrafish could be an alternative model to study the molecular pathways of this disease as well as to test possible therapeutic approaches capable of rescuing the phenotype.
非典型瑞特综合征是一种由CDKL5基因突变引起的儿童神经发育障碍,其特征是发育逐渐退化,出现手部目的性使用丧失、脑和头部生长减缓、行走问题、癫痫发作以及智力残疾。目前,这种病症无法治愈,关于能够模拟其表型的动物模型的信息也很少,因此开发更多的动物模型对于深入了解该疾病应该是有意义的。斑马鱼已成功用作许多人类遗传疾病的模式生物;然而,关于该生物中cdkl5直系同源基因的时空表达尚无信息。在本研究中,我们通过定量PCR和全胚胎原位杂交确定了斑马鱼中cdkl5的发育表达模式。cdkl5在发育的最初24小时内以低水平母源表达。此后,该基因的表达显著增加,并且开始主要在神经系统以及几个脑结构中表达,如端脑、中脑和间脑。斑马鱼中cdkl5的表达模式与已知在人类中受影响的组织一致,并与瑞特综合征患者中观察到的症状和缺陷相关,从而提供了首个证据,表明斑马鱼可能是研究该疾病分子途径以及测试能够挽救表型的可能治疗方法的替代模型。