huHSD17B13 Mouse
製品名
huHSD17B13 Mouse
製品ID
C002014
系統名
C57BL/6JCya-Hsd17b13tm1(hHSD17B13)/Cya
背景情報
C57BL/6JCya
状況
このマウス系統を論文で使用する場合は、「huHSD17B13 Mouse（カタログ番号C002014）はサイアジェンから購入しました。」と引用してください。
製品タイプ
年齢
遺伝子型
性別
数量
遺伝子名
遺伝子別名
FLDP, SCDR9, NIIL497, SDR16C3, HMFN0376
NCBI ID
染色体
Chr 4
MGI ID
さらに
系統詳細
17β-hydroxysteroid dehydrogenase 13 (HSD17B13) is a liver-enriched lipid droplet (LD)-associated protein that catalyzes the interconversion between 17-ketosteroids and 17-hydroxysteroids. HSD17B13 protein is selectively expressed in liver cells and localizes exclusively to the surface of lipid droplets, with similar tissue distribution and subcellular localization observed in both humans and mice [1]. HSD17B13 may play a significant role in regulating the biogenesis, growth, and degradation of lipid droplets in the liver. Metabolic-associated fatty liver disease (MAFLD) is pathologically defined by abnormal accumulation of neutral lipids (such as triglycerides and cholesterol esters) within hepatocellular lipid droplets. Its inflammatory form, metabolic-associated steatohepatitis (MASH), is closely associated with the pathogenesis of chronic liver diseases. Research has found that HSD17B13 protein expression is markedly elevated in the livers of MAFLD patients compared to healthy individuals [2-3]. Abnormal expression and dysfunction of HSD17B13 may be one of the pathogenic mechanisms of chronic liver disease, particularly in the MAFLD. As a potential therapeutic target for MAFLD and MASH, there are currently clinical-stage drug pipelines targeting the HSD17B13 gene, including ASO drug ION-455 developed by Ionis and siRNA drug Rapirosiran developed by Regeneron Pharmaceuticals.
The huHSD17B13 mouse is a humanized model established by gene editing. The sequences from the ATG start codon to the TAG stop codon of the endogenous mouse Hsd17b13 gene were replaced with the sequences from the ATG start codon to the TGA stop codon of the human HSD17B13 gene. This model is suitable for investigating the pathogenic mechanism of the HSD17B13 gene in metabolic dysfunction-associated fatty liver disease and related chronic liver diseases, as well as for the development and efficacy evaluation of targeted drugs.
参考文献
Zhang HB, Su W, Xu H, Zhang XY, Guan YF. HSD17B13: A Potential Therapeutic Target for NAFLD. Front Mol Biosci. 2022 Jan 7;8:824776.
Su W, Wang Y, Jia X, Wu W, Li L, Tian X, Li S, Wang C, Xu H, Cao J, Han Q, Xu S, Chen Y, Zhong Y, Zhang X, Liu P, Gustafsson JÅ, Guan Y. Comparative proteomic study reveals 17β-HSD13 as a pathogenic protein in nonalcoholic fatty liver disease. Proc Natl Acad Sci U S A. 2014 Aug 5;111(31):11437-42.
Ma Y, Belyaeva OV, Brown PM, Fujita K, Valles K, Karki S, de Boer YS, Koh C, Chen Y, Du X, Handelman SK, Chen V, Speliotes EK, Nestlerode C, Thomas E, Kleiner DE, Zmuda JM, Sanyal AJ; (for the Nonalcoholic Steatohepatitis Clinical Research Network); Kedishvili NY, Liang TJ, Rotman Y. 17-Beta Hydroxysteroid Dehydrogenase 13 Is a Hepatic Retinol Dehydrogenase Associated With Histological Features of Nonalcoholic Fatty Liver Disease. Hepatology. 2019 Apr;69(4):1504-1519.
系統作製戦略
The sequences from the ATG start codon to the TAG stop codon of the endogenous mouse Hsd17b13 gene were replaced with the sequences from the ATG start codon to the TGA stop codon of the human HSD17B13 gene.
Figure 1. Gene editing strategy for huHSD17B13 mice.
適用分野
Research on metabolism-associated fatty liver disease (MAFLD), metabolism-associated steatohepatitis (MASH), and other chronic liver diseases;
Preclinical evaluation of HSD17B13-targeted drugs.
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