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原发性辅酶 Q 缺乏症:文献回顾和临床特征在线平台,以揭示基因型-表型相关性。

Primary Coenzyme Q deficiencies: A literature review and online platform of clinical features to uncover genotype-phenotype correlations.

机构信息

Centro Andaluz de Biología del Desarrollo, Universidad Pablo de Olavide-CSIC-JA and CIBERER, Instituto de Salud Carlos III, Seville, 41013, Spain.

Departamento de Biología Vegetal y Ecología, Universidad de Sevilla, Seville, 41012, Spain.

出版信息

Free Radic Biol Med. 2021 May 1;167:141-180. doi: 10.1016/j.freeradbiomed.2021.02.046. Epub 2021 Mar 4.

Abstract

Primary Coenzyme Q (CoQ) deficiencies are clinically heterogeneous conditions and lack clear genotype-phenotype correlations, complicating diagnosis and prognostic assessment. Here we present a compilation of all the symptoms and patients with primary CoQ deficiency described in the literature so far and analyse the most common clinical manifestations associated with pathogenic variants identified in the different COQ genes. In addition, we identified new associations between the age of onset of symptoms and different pathogenic variants, which could help to a better diagnosis and guided treatment. To make these results useable for clinicians, we created an online platform (https://coenzymeQbiology.github.io/clinic-CoQ-deficiency) about clinical manifestations of primary CoQ deficiency that will be periodically updated to incorporate new information published in the literature. Since CoQ primary deficiency is a rare disease, the available data are still limited, but as new patients are added over time, this tool could become a key resource for a more efficient diagnosis of this pathology.

摘要

原发性辅酶 Q(CoQ)缺乏症是临床异质性疾病,缺乏明确的基因型-表型相关性,这使得诊断和预后评估变得复杂。在这里,我们汇集了迄今为止文献中描述的所有原发性 CoQ 缺乏症的症状和患者,并分析了与不同 COQ 基因中发现的致病性变异相关的最常见临床表现。此外,我们还确定了症状发作年龄与不同致病性变异之间的新关联,这有助于更好的诊断和指导治疗。为了使这些结果对临床医生有用,我们创建了一个关于原发性 CoQ 缺乏症临床表现的在线平台(https://coenzymeQbiology.github.io/clinic-CoQ-deficiency),该平台将定期更新,纳入文献中发表的新信息。由于 CoQ 原发性缺乏症是一种罕见疾病,因此可用数据仍然有限,但随着时间的推移,随着新患者的加入,该工具可能成为更有效地诊断这种病理的关键资源。

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