编码佩瓦金(一种新发现的传入听觉通路蛋白)的基因突变会导致DFNB59型听觉神经病。

Mutations in the gene encoding pejvakin, a newly identified protein of the afferent auditory pathway, cause DFNB59 auditory neuropathy.

作者信息

Delmaghani Sedigheh, del Castillo Francisco J, Michel Vincent, Leibovici Michel, Aghaie Asadollah, Ron Uri, Van Laer Lut, Ben-Tal Nir, Van Camp Guy, Weil Dominique, Langa Francina, Lathrop Mark, Avan Paul, Petit Christine

机构信息

Unité de Génétique des Déficits Sensoriels INSERM U587, Institut Pasteur, 25, rue du Docteur Roux, 75724 Paris Cedex 15, France.

出版信息

Nat Genet. 2006 Jul;38(7):770-8. doi: 10.1038/ng1829. Epub 2006 Jun 25.

Abstract

Auditory neuropathy is a particular type of hearing impairment in which neural transmission of the auditory signal is impaired, while cochlear outer hair cells remain functional. Here we report on DFNB59, a newly identified gene on chromosome 2q31.1-q31.3 mutated in four families segregating autosomal recessive auditory neuropathy. DFNB59 encodes pejvakin, a 352-residue protein. Pejvakin is a paralog of DFNA5, a protein of unknown function also involved in deafness. By immunohistofluorescence, pejvakin is detected in the cell bodies of neurons of the afferent auditory pathway. Furthermore, Dfnb59 knock-in mice, homozygous for the R183W variant identified in one DFNB59 family, show abnormal auditory brainstem responses indicative of neuronal dysfunction along the auditory pathway. Unlike previously described sensorineural deafness genes, all of which underlie cochlear cell pathologies, DFNB59 is the first human gene implicated in nonsyndromic deafness due to a neuronal defect.

摘要

听神经病是一种特殊类型的听力障碍,其中听觉信号的神经传递受损,而耳蜗外毛细胞仍保持功能。在此我们报告DFNB59,这是一个新鉴定出的位于2号染色体2q31.1 - q31.3区域的基因,在四个常染色体隐性遗传听神经病家系中发生突变。DFNB59编码佩瓦金蛋白,一种由352个氨基酸残基组成的蛋白质。佩瓦金蛋白是DFNA5的旁系同源物,DFNA5是一种功能未知但也与耳聋有关的蛋白质。通过免疫荧光法检测发现,佩瓦金蛋白存在于传入听觉通路神经元的细胞体中。此外,在一个DFNB59家系中鉴定出的R183W变异的纯合Dfnb59基因敲入小鼠,表现出异常的听觉脑干反应,表明听觉通路上存在神经元功能障碍。与先前描述的所有导致耳蜗细胞病变的感音神经性耳聋基因不同,DFNB59是首个因神经元缺陷导致非综合征性耳聋的人类基因。

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