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鉴定秀丽隐杆线虫中 BSCL2 脂肪营养不良致病变体的遗传抑制因子。

Identification of genetic suppressors for a BSCL2 lipodystrophy pathogenic variant in Caenorhabditis elegans.

机构信息

Department of Biology, University of Florida, Gainesville, FL 32610, USA.

Genetics Institute, University of Florida, Gainesville, FL 32610, USA.

出版信息

Dis Model Mech. 2024 Jun 1;17(6). doi: 10.1242/dmm.050524. Epub 2024 Apr 16.

Abstract

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.

摘要

Seipin(BSCL2)是一种保守的内质网蛋白,在脂滴(LD)的生物发生和调节 LD 形态中起着关键作用,其致病性变体与 Berardinelli-Seip 先天性全身性脂肪营养不良 2 型(BSCL2)有关。为了模拟 BSCL2 疾病,我们在秀丽隐杆线虫中生成了一个同源的 BSCL2 变体 seip-1(A185P)。在这项研究中,我们进行了一项无偏化学诱变筛选,以鉴定能够恢复 seip-1(A185P)突变体背景下胚胎活力的遗传抑制子。从筛选中总共分离并回收了 5 个抑制子系。seip-1(A185P)的缺陷表型,包括胚胎致死和卵壳形成受损,在每个抑制子系中都得到了显著抑制。这 5 个抑制子系中的两个也缓解了卵母细胞中 LD 的增大。然后,我们定位了一个抑制子候选基因 lmbr-1,它是人类肢体发育膜蛋白 1(LMBR1)的同源物。CRISPR/Cas9 编辑的 lmbr-1 抑制子等位基因 lmbr-1(S647F)和 lmbr-1(P314L),均显著抑制了 seip-1(A185P)背景下的胚胎致死和卵壳形成缺陷。新鉴定的抑制子系为研究可能在脂肪营养不良模型中调节 seipin 的潜在遗传相互作用子和途径提供了有价值的见解。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/987f/11051982/55ffd14829ca/dmm-17-050524-g1.jpg

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