Suppr超能文献

当代涉及癫痫或发作的代谢性先天缺陷的范围。

Contemporary scope of inborn errors of metabolism involving epilepsy or seizures.

机构信息

Institute of Biomedical Sciences, Faculty of Medicine, Vilnius University, Santariskiu 2, LT-08661, Vilnius, Lithuania.

Vilnius University Hospital Santaros Klinikos, Santariskiu 2, LT-08661, Vilnius, Lithuania.

出版信息

Metab Brain Dis. 2018 Dec;33(6):1781-1786. doi: 10.1007/s11011-018-0288-1. Epub 2018 Jul 13.

Abstract

Many inborn errors of metabolism may present with epilepsy or seizures, however, current scope of these diseases is unknown. Due to available precision medicine approaches in many inborn errors of metabolism and sophisticated traditional diagnostics, this group of disorders is of special relevance to clinicians. Besides, as current treatment is challenging and unsuccessful in more than 30% of all epilepsy patients, these diseases may provide valuable models for ictogenesis and epileptogenesis studies and potentially pave the ways to identification of novel treatments. The aim of this study was to elucidate genetic architecture of inborn errors of metabolism involving epilepsy or seizures and to evaluate their diagnostic approaches. After extensive search, 880 human genes were identified with a considerable part, 373 genes (42%), associated with inborn errors of metabolism. The most numerous group comprised disorders of energy metabolism (115, 31% of all inborn errors of metabolism). A substantial number of these diseases (26%, 97/373) have established specific treatments, therefore timely diagnosis comes as an obligation. Highly heterogenous, overlapping and non-specific phenotypes in most of inborn errors of metabolism presenting with epilepsy or seizures usually preclude phenotype-driven diagnostics. Besides, as traditional diagnostics involves a range of specialized metabolic tests with low diagnostic yields and is generally inefficient and lengthy, next-generation sequencing-based methods were proposed as a cost-efficient one-step way to shorten "diagnostic odyssey". Extensive list of 373 epilepsy- or seizures-associated inborn errors of metabolism genes may be of value in development of gene panels and as a tool for variants' filtration.

摘要

许多先天性代谢错误可能表现为癫痫或癫痫发作,但目前这些疾病的范围尚不清楚。由于许多先天性代谢错误中存在可用的精准医学方法和复杂的传统诊断方法,因此这些疾病对临床医生具有特殊意义。此外,由于目前的治疗方法在超过 30%的所有癫痫患者中都具有挑战性和不成功,因此这些疾病可能为癫痫发生和癫痫形成研究提供有价值的模型,并有可能为新的治疗方法铺平道路。本研究旨在阐明涉及癫痫或癫痫发作的先天性代谢错误的遗传结构,并评估其诊断方法。经过广泛搜索,确定了 880 个人类基因,其中相当一部分(373 个基因,占 42%)与先天性代谢错误有关。数量最多的一组是能量代谢紊乱(115 种,占所有先天性代谢错误的 31%)。这些疾病中有相当一部分(26%,97/373)已经建立了特定的治疗方法,因此及时诊断是必要的。大多数表现为癫痫或癫痫发作的先天性代谢错误具有高度异质性、重叠性和非特异性表型,通常排除了基于表型的诊断。此外,由于传统诊断方法涉及一系列具有低诊断率的专门代谢测试,并且通常效率低下且冗长,因此基于下一代测序的方法被提议作为一种具有成本效益的一步缩短“诊断探索”的方法。广泛的 373 个与癫痫或癫痫发作相关的先天性代谢错误基因列表可能有助于开发基因面板,并作为变异过滤的工具。

文献AI研究员

20分钟写一篇综述,助力文献阅读效率提升50倍。

立即体验

用中文搜PubMed

大模型驱动的PubMed中文搜索引擎

马上搜索

文档翻译

学术文献翻译模型,支持多种主流文档格式。

立即体验