鉴定草履虫 BSCL2 脂肪营养不良致病变体的基因抑制因子

IF 4 3区 医学 Q2 CELL BIOLOGY
Disease Models & Mechanisms Pub Date : 2024-06-01 Epub Date: 2024-04-16 DOI:10.1242/dmm.050524
Xiaofei Bai, Harold E Smith, Andy Golden
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引用次数: 0

摘要

Seipin(BSCL2)是一种保守的内质网蛋白,在低密度脂蛋白生物发生和调节低密度脂蛋白形态方面起着关键作用,其致病变体与 Berardinelli-Seip 先天性全身脂肪营养不良 2 型(BSCL2)有关。为了模拟 BSCL2 疾病,我们在秀丽隐杆线虫中产生了与 BSCL2 变体 seip-1(A185P) 同源的变体。在这项研究中,我们进行了无偏见的化学诱变筛选,以鉴定能在 seip-1(A185P)突变体背景下恢复胚胎活力的基因抑制因子。筛选共分离并恢复了五个抑制株系。每个抑制系都显著抑制了seip-1(A185P)的缺陷表型,包括胚胎致死和蛋壳形成受损。五个抑制系中的两个也减轻了卵母细胞中LD的扩大。我们随后绘制了一个候选抑制基因 lmbr-1,它是人类 LMBR1(肢体发育膜蛋白 1)的直系同源物。CRISPR/Cas9编辑的lmbr-1抑制等位基因lmbr-1(Ser647Phe)和lmbr-1(Pro314Leu)都显著抑制了seip-1(A185P)背景下的胚胎致死率和蛋壳形成缺陷。新发现的抑制系为了解脂肪营养不良模型中可能调控seipin的潜在遗传相互作用因子和途径提供了宝贵的见解。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Identification of genetic suppressors for a BSCL2 lipodystrophy pathogenic variant in Caenorhabditis elegans.

Seipin (BSCL2), a conserved endoplasmic reticulum protein, plays a critical role in lipid droplet (LD) biogenesis and in regulating LD morphology, pathogenic variants of which are associated with Berardinelli-Seip congenital generalized lipodystrophy type 2 (BSCL2). To model BSCL2 disease, we generated an orthologous BSCL2 variant, seip-1(A185P), in Caenorhabditis elegans. In this study, we conducted an unbiased chemical mutagenesis screen to identify genetic suppressors that restore embryonic viability in the seip-1(A185P) mutant background. A total of five suppressor lines were isolated and recovered from the screen. The defective phenotypes of seip-1(A185P), including embryonic lethality and impaired eggshell formation, were significantly suppressed in each suppressor line. Two of the five suppressor lines also alleviated the enlarged LDs in the oocytes. We then mapped a suppressor candidate gene, lmbr-1, which is an ortholog of human limb development membrane protein 1 (LMBR1). The CRISPR/Cas9 edited lmbr-1 suppressor alleles, lmbr-1(S647F) and lmbr-1(P314L), both significantly suppressed embryonic lethality and defective eggshell formation in the seip-1(A185P) background. The newly identified suppressor lines offer valuable insights into potential genetic interactors and pathways that may regulate seipin in the lipodystrophy model.

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来源期刊
Disease Models & Mechanisms
Disease Models & Mechanisms 医学-病理学
CiteScore
6.60
自引率
7.00%
发文量
203
审稿时长
6-12 weeks
期刊介绍: Disease Models & Mechanisms (DMM) is an online Open Access journal focusing on the use of model systems to better understand, diagnose and treat human disease.
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