一名共济性脑瘫患者Joubert综合征和听力损害的分子评价。

IF 1.2 Q4 GENETICS & HEREDITY Global Medical Genetics Pub Date : 2023-09-01 DOI:10.1055/s-0043-1771184
N Sreedevi, N Swapna, Santosh Maruthy, T Jayakumar, Charles Sylvester
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摘要

Joubert综合征(JBTS)是一种罕见的常染色体隐性遗传或x连锁的先天性脑畸形,具有很强的遗传异质性。JBTS的其他神经学特征包括张力减退、共济失调、发育迟缓和认知障碍。JBTS导致的听力损失在文献中已有报道。我们提出一个3.5岁的男孩出生在一个健康的近亲南印度夫妇谁提出了共济性脑瘫(CP)和听力障碍的情况下;医学报告证实了JBTS典型的脑部畸形。听力损害通过听力学评估筛查,证实存在严重的深度听力损失和外毛细胞功能障碍。进行全外显子组测序(WES)以了解病情的分子方面并检测任何新的突变。鉴定出与JBTS 3型相关的纯合突变ahi1c . 2023g > A和与听力障碍相关的GJB2 c.71G > A。Sanger测序验证结果,在患者父母中鉴定出杂合子AHI1 c.2023G > A和GJB2 c.71G > A。本研究证实,通过WES诊断JBTS有助于确定遗传性疾病的遗传原因,从而加快对高危家庭的遗传评估和咨询。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

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Molecular Evaluation of Joubert Syndrome and Hearing Impairment in a Patient with Ataxic Cerebral Palsy.

Joubert syndrome (JBTS) is a rare autosomal recessive or X-linked congenital brain malformation with strong genetic heterogeneity. Other neurological features of JBTS include hypotonia, ataxia, developmental delay, and cognitive impairment. Hearing loss with JBTS has been reported in the literature. We present the case of a 3.5-year-old boy born to a healthy consanguineous South Indian couple who was presented with ataxic cerebral palsy (CP) and hearing impairment; medical reports confirmed typical brain malformations of JBTS. Hearing impairment was screened by audiological assessment, which confirmed the presence of severe-profound hearing loss with outer hair cell dysfunction. Whole-exome sequencing (WES) was performed to know the molecular aspects of the condition and to detect any novel mutations. The homozygous mutation AHI1 c.2023G > A associated with JBTS type 3 and GJB2 c.71G > A mutation associated with hearing impairment were identified. Sanger sequencing was performed to validate the result and it identified heterozygous AHI1 c.2023G > A and GJB2 c.71G > A in the patient's parents. This study confirms the diagnosis of JBTS by WES helps identify the genetic causes of hereditary disorders that accelerate genetic evaluation and counseling for at-risk families.

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来源期刊
Global Medical Genetics
Global Medical Genetics GENETICS & HEREDITY-
自引率
11.80%
发文量
30
审稿时长
14 weeks
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