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Cou Cou, flying fish and a whole exome please... lessons learned from genetic testing in Barbados.库库,飞鱼和全外显子组......巴巴多斯基因检测的经验教训。
Pan Afr Med J. 2021 Feb 3;38:111. doi: 10.11604/pamj.2021.38.111.27969. eCollection 2021.
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本文引用的文献

1
A brief history of human disease genetics.人类疾病遗传学简史。
Nature. 2020 Jan;577(7789):179-189. doi: 10.1038/s41586-019-1879-7. Epub 2020 Jan 8.
2
The burden of rare diseases.罕见病的负担。
Am J Med Genet A. 2019 Jun;179(6):885-892. doi: 10.1002/ajmg.a.61124. Epub 2019 Mar 18.
3
empowering the neurogenetic testing services in developing countries: use the basic skills with speed and scale.增强发展中国家的神经遗传学检测服务:快速且大规模地运用基本技能。
Ann Neurosci. 2015 Jan;22(1):1-3. doi: 10.5214/ans.0972.7531.220101.
4
The National Institutes of Health Undiagnosed Diseases Program: insights into rare diseases.美国国立卫生研究院不明疾病计划:罕见病的研究进展。
Genet Med. 2012 Jan;14(1):51-9. doi: 10.1038/gim.0b013e318232a005. Epub 2011 Sep 26.

库库,飞鱼和全外显子组......巴巴多斯基因检测的经验教训。

Cou Cou, flying fish and a whole exome please... lessons learned from genetic testing in Barbados.

机构信息

Departments of Pediatrics and Clinical Neuroscience, Cumming School of Medicine, University of Calgary, Calgary, Alberta, Canada.

Queen Elizabeth Hospital, Bridgetown, Barbados.

出版信息

Pan Afr Med J. 2021 Feb 3;38:111. doi: 10.11604/pamj.2021.38.111.27969. eCollection 2021.

DOI:10.11604/pamj.2021.38.111.27969
PMID:33912281
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC8051213/
Abstract

Millions of patients, with suspected complex neurogenetic disorders, living in resource limited regions around the world have no access to genetic testing despite the rapidly expanding availability and decreasing costs of genetic testing in first world nations. The barriers to increasing availability of genetic testing in resource limited nations are multifactorial but can be attributed, in large part, to a lack of awareness of the power of genetic testing to lead to a rapid, cost-effective, diagnosis that potentially will have profound clinical implications on treatment and patient outcomes. We report our experience with whole exome sequencing (WES) done for the first time in 5 patients of African descent with a suspected neurogenetic disorder living in a resource limited setting on the Eastern Caribbean island of Barbados. A diagnostic pathogenic mutation was found in 3 patients in the SCN1A, STXBP1 and SCN4A, who clinically were diagnosed with Dravet syndrome, Lennox-Gastaut syndrome, paramytonia and seizures respectively. A variant of undetermined significance was found in a patient with global developmental delays, hypotonia, with abnormal eye movements. In one patient WES was non-diagnostic. This result highlights the high yield of WES in carefully selected patients with a neurologic disease and the need for increase access to genetic testing in resource limited settings globally.

摘要

尽管在第一世界国家,基因检测的可及性迅速扩大,成本不断降低,但全球范围内资源有限地区仍有数百万名疑似复杂神经遗传疾病的患者无法接受基因检测。在资源有限的国家增加基因检测可及性的障碍是多方面的,但在很大程度上可以归因于缺乏对基因检测能够快速、具有成本效益地进行诊断的认识,这种诊断可能对治疗和患者预后产生深远的临床影响。我们报告了在巴巴多斯(位于东加勒比海)这样一个资源有限的环境中,首次对 5 名非洲裔疑似神经遗传疾病患者进行全外显子组测序(WES)的经验。在 3 名 SCN1A、STXBP1 和 SCN4A 致病性突变的患者中发现了诊断性致病突变,他们分别被临床诊断为 Dravet 综合征、Lennox-Gastaut 综合征、肌强直和癫痫发作。一名患有全面发育迟缓、张力减退和眼球运动异常的患者发现了一种意义未明的变异。在一名患者中,WES 检测结果无法诊断。这一结果突显了 WES 在精心挑选的神经疾病患者中的高检出率,以及在全球范围内增加资源有限地区基因检测可及性的必要性。