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Smith-Lemli-Opitz 综合征的听觉表型。

Auditory phenotype of Smith-Lemli-Opitz syndrome.

机构信息

NIDCD, National Institutes of Health, Bethesda, Maryland, USA.

Cleveland Clinic Head & Neck Institute, Cleveland, Ohio, USA.

出版信息

Am J Med Genet A. 2021 Apr;185(4):1131-1141. doi: 10.1002/ajmg.a.62087. Epub 2021 Feb 2.

DOI:10.1002/ajmg.a.62087
PMID:33529473
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC7994936/
Abstract

Smith-Lemli-Opitz syndrome (SLOS) is an autosomal recessive multiple congenital malformation and intellectual disability syndrome resulting from variants in DHCR7. Auditory characteristics of persons with SLOS have been described in limited case reports but have not been systematically evaluated. The objective of this study is to describe the auditory phenotype in SLOS. Age- and ability-appropriate hearing evaluations were conducted on 32 patients with SLOS. A subset of 21 had auditory brainstem response testing, from which an auditory neural phenotype is described. Peripheral or retrocochlear auditory dysfunction was observed in at least one ear of 65.6% (21) of the patients in our SLOS cohort. The audiometric phenotype was heterogeneous and included conductive, mixed, and sensorineural hearing loss. The most common presentation was a slight to mild conductive hearing loss, although profound sensorineural hearing loss was also observed. Abnormal auditory brainstem responses indicative of retrocochlear dysfunction were identified in 21.9% of the patients. Many were difficult to test behaviorally and required objective assessment methods to estimate hearing sensitivity. Individuals with SLOS are likely to have hearing loss that may impact communication, including speech and language development. Routine audiologic surveillance should be conducted to ensure prompt management of hearing loss.

摘要

史密斯-莱姆利-奥皮茨综合征(SLOS)是一种常染色体隐性遗传的多发先天畸形和智力障碍综合征,由 DHCR7 变异引起。已有有限的病例报告描述了 SLOS 患者的听觉特征,但尚未进行系统评估。本研究旨在描述 SLOS 的听觉表型。对 32 名 SLOS 患者进行了年龄和能力适当的听力评估。其中 21 名患者进行了听觉脑干反应测试,从中描述了听觉神经表型。我们的 SLOS 队列中至少有 65.6%(21 名)的患者在一只或双耳中观察到外周或耳蜗后听觉功能障碍。听力学表型呈异质性,包括传导性、混合性和感音神经性听力损失。最常见的表现为轻度至中度传导性听力损失,但也观察到重度感音神经性听力损失。在 21.9%的患者中发现了提示耳蜗后功能障碍的异常听觉脑干反应。许多患者难以进行行为测试,需要使用客观评估方法来估计听力敏感度。SLOS 患者可能存在听力损失,这可能会影响交流,包括言语和语言发育。应进行常规听力监测,以确保及时管理听力损失。

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Auditory Phenotype of Smith-Magenis Syndrome.史密斯-马吉尼斯综合征的听觉表型
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Normal IQ is possible in Smith-Lemli-Opitz syndrome.史密斯-勒米-奥皮茨综合征患者有可能拥有正常智商。
Am J Med Genet A. 2017 Aug;173(8):2097-2100. doi: 10.1002/ajmg.a.38125. Epub 2017 Mar 27.
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Smith-Lemli-Opitz syndrome carrier frequency and estimates of in utero mortality rates.史密斯-勒米-奥皮茨综合征携带者频率及宫内死亡率估计
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Endogenous B-ring oxysterols inhibit the Hedgehog component Smoothened in a manner distinct from cyclopamine or side-chain oxysterols.内源性B环氧化甾醇以一种不同于环杷明或侧链氧化甾醇的方式抑制Hedgehog信号通路组件Smoothened。
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Modeling Smith-Lemli-Opitz syndrome with induced pluripotent stem cells reveals a causal role for Wnt/β-catenin defects in neuronal cholesterol synthesis phenotypes.利用诱导多能干细胞对史密斯-勒米-奥皮茨综合征进行建模,揭示了Wnt/β-连环蛋白缺陷在神经元胆固醇合成表型中的因果作用。
Nat Med. 2016 Apr;22(4):388-96. doi: 10.1038/nm.4067. Epub 2016 Mar 21.
7
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