一项最新的氧化磷酸化必需基因清单确定了家族性mims病的线粒体起源。

IF 6.9 1区 生物学 Q1 CELL BIOLOGY
Marcell Harhai, Mads M Foged, Christine Zarges, Juan C Landoni, Sylvain Chollet, Michele Simonelli, Emeline Recazens, Miriam Lisci, Nora Laban, Suliana Manley, Jan Riemer, Jose Antonio Lopez-Escamez, Anna Lysakowski, Alexis A Jourdain
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引用次数: 0

摘要

线粒体疾病(MDs)是最常见的先天性代谢错误之一,氧化磷酸化(OXPHOS)功能障碍是一个标志。其复杂的遗传模式和多样的临床表现使得MDs的诊断具有挑战性,并且迄今为止,大多数MDs缺乏治愈方法。在此,我们建立在先前鉴定OXPHOS所需基因的基础上,并报告了一个高度互补的半乳糖致敏CRISPR-Cas9“生长”筛选,展示了481个OXPHOS基因的最新清单,其中包括157个与MDs相关的基因。我们进一步关注FAM136A,这是一种与msamuire病相关的基因,并证明它支持细胞系、小鼠和患者的膜间空间蛋白稳态和OXPHOS。我们的研究确定了家族性mims疾病的线粒体基础,提供了oxphos相关基因的综合资源,并阐明了MDs涉及的途径,具有指导未来MDs诊断和治疗的潜力。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
An updated inventory of genes essential for oxidative phosphorylation identifies a mitochondrial origin in familial Ménière's disease.

Mitochondrial disorders (MDs) are among the most common inborn errors of metabolism, and dysfunction in oxidative phosphorylation (OXPHOS) is a hallmark. Their complex mode of inheritance and diverse clinical presentations render the diagnosis of MDs challenging, and, to date, most lack a cure. Here, we build on previous efforts to identify genes necessary for OXPHOS and report a highly complementary galactose-sensitized CRISPR-Cas9 "growth" screen, presenting an updated inventory of 481 OXPHOS genes, including 157 linked to MDs. We further focus on FAM136A, a gene associated with Ménière's disease, and demonstrate that it supports intermembrane space protein homeostasis and OXPHOS in cell lines, mice, and patients. Our study identifies a mitochondrial basis in familial Ménière's disease, provides a comprehensive resource of OXPHOS-related genes, and sheds light on the pathways involved in MDs, with the potential to guide future diagnostics and treatments for MDs.

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来源期刊
Cell reports
Cell reports CELL BIOLOGY-
CiteScore
13.80
自引率
1.10%
发文量
1305
审稿时长
77 days
期刊介绍: Cell Reports publishes high-quality research across the life sciences and focuses on new biological insight as its primary criterion for publication. The journal offers three primary article types: Reports, which are shorter single-point articles, research articles, which are longer and provide deeper mechanistic insights, and resources, which highlight significant technical advances or major informational datasets that contribute to biological advances. Reviews covering recent literature in emerging and active fields are also accepted. The Cell Reports Portfolio includes gold open-access journals that cover life, medical, and physical sciences, and its mission is to make cutting-edge research and methodologies available to a wide readership. The journal's professional in-house editors work closely with authors, reviewers, and the scientific advisory board, which consists of current and future leaders in their respective fields. The advisory board guides the scope, content, and quality of the journal, but editorial decisions are independently made by the in-house scientific editors of Cell Reports.
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