Parisi Melissa A, Doherty Dan, Chance Phillip F, Glass Ian A
Division of Genetics and Developmental Medicine, Department of Pediatrics, University of Washington School of Medicine, Seattle, WA 98195-6320, USA.
Eur J Hum Genet. 2007 May;15(5):511-21. doi: 10.1038/sj.ejhg.5201648. Epub 2007 Mar 21.
Joubert syndrome (JS) and related disorders are characterized by the 'molar tooth sign' (cerebellar vermis hypoplasia and brainstem anomalies) on MRI, hypotonia, developmental delay, ataxia, irregular breathing pattern and abnormal eye movements. Combinations of additional features such as polydactyly, ocular coloboma, retinal dystrophy, renal disease, hepatic fibrosis, encephalocele, and other brain malformations define clinical sub-types. Recent identification of the NPHP1, AHI1, and CEP290 genes has started to reveal the molecular basis of JS, which may implicate the primary cilium in these disorders. Additional genes remain to be identified.
Joubert综合征(JS)及相关疾病的特征为MRI上出现“磨牙征”(小脑蚓部发育不全和脑干异常)、肌张力减退、发育迟缓、共济失调、呼吸模式不规则及眼球运动异常。多指(趾)畸形、眼裂、视网膜营养不良、肾病、肝纤维化、脑膨出及其他脑畸形等附加特征的组合可定义临床亚型。最近对NPHP1、AHI1和CEP290基因的鉴定已开始揭示JS的分子基础,这可能表明这些疾病中存在原发性纤毛。仍有待鉴定其他基因。