Muggenthaler Martina M A, Chowdhury Biswajit, Hasan S Naimul, Cross Harold E, Mark Brian, Harlalka Gaurav V, Patton Michael A, Ishida Miho, Behr Elijah R, Sharma Sanjay, Zahka Kenneth, Faqeih Eissa, Blakley Brian, Jackson Mike, Lees Melissa, Dolinsky Vernon, Cross Leroy, Stanier Philip, Salter Claire, Baple Emma L, Alkuraya Fowzan S, Crosby Andrew H, Triggs-Raine Barbara, Chioza Barry A
RILD Wellcome Wolfson Centre, University of Exeter Medical School, Exeter, United Kingdom.
Department of Biochemistry & Medical Genetics, University of Manitoba, Winnipeg, Manitoba, Canada.
PLoS Genet. 2017 Jan 12;13(1):e1006470. doi: 10.1371/journal.pgen.1006470. eCollection 2017 Jan.
Orofacial clefting is amongst the most common of birth defects, with both genetic and environmental components. Although numerous studies have been undertaken to investigate the complexities of the genetic etiology of this heterogeneous condition, this factor remains incompletely understood. Here, we describe mutations in the HYAL2 gene as a cause of syndromic orofacial clefting. HYAL2, encoding hyaluronidase 2, degrades extracellular hyaluronan, a critical component of the developing heart and palatal shelf matrix. Transfection assays demonstrated that the gene mutations destabilize the molecule, dramatically reducing HYAL2 protein levels. Consistent with the clinical presentation in affected individuals, investigations of Hyal2-/- mice revealed craniofacial abnormalities, including submucosal cleft palate. In addition, cor triatriatum sinister and hearing loss, identified in a proportion of Hyal2-/- mice, were also found as incompletely penetrant features in affected humans. Taken together our findings identify a new genetic cause of orofacial clefting in humans and mice, and define the first molecular cause of human cor triatriatum sinister, illustrating the fundamental importance of HYAL2 and hyaluronan turnover for normal human and mouse development.
口面部裂隙是最常见的出生缺陷之一,具有遗传和环境因素。尽管已经进行了大量研究来探究这种异质性疾病遗传病因的复杂性,但这一因素仍未被完全理解。在此,我们描述了HYAL2基因突变是综合征性口面部裂隙的一个病因。HYAL2编码透明质酸酶2,可降解细胞外透明质酸,而透明质酸是发育中的心脏和腭板基质的关键成分。转染实验表明,基因突变使该分子不稳定,显著降低了HYAL2蛋白水平。与受影响个体的临床表现一致,对Hyal2-/-小鼠的研究揭示了颅面异常,包括黏膜下腭裂。此外,在一部分Hyal2-/-小鼠中发现的左心三房心和听力损失,在受影响的人类中也被发现为不完全显性特征。综合我们的研究结果,确定了人类和小鼠口面部裂隙的一个新的遗传病因,并确定了人类左心三房心的首个分子病因,说明了HYAL2和透明质酸代谢对正常人类和小鼠发育的根本重要性。