Kawasaki Haruhisa, Suzuki Takahiro, Ito Kumpei, Takahara Tsubasa, Goto-Inoue Naoko, Setou Mitsutoshi, Sakata Kazuki, Ishida Norio
Foundation for Advancement of International Science, 24-16, Kasuga, 3-chome, Tsukuba, Ibaraki 305-0821, Japan; Ishida Group of Clock Gene, National Institute of Advanced Industrial Science and Technology, Tsukuba, Ibaraki 305-8566, Japan.
Foundation for Advancement of International Science, 24-16, Kasuga, 3-chome, Tsukuba, Ibaraki 305-0821, Japan; Ishida Group of Clock Gene, National Institute of Advanced Industrial Science and Technology, Tsukuba, Ibaraki 305-8566, Japan; Shigray Inc., Tsuchiura, Ibaraki 300-0876, Japan.
Gene. 2017 May 30;614:49-55. doi: 10.1016/j.gene.2017.03.004. Epub 2017 Mar 7.
Gaucher's disease in humans is considered a deficiency of glucocerebrosidase (GlcCerase) that result in the accumulation of its substrate, glucocerebroside (GlcCer). Although mouse models of Gaucher's disease have been reported from several laboratories, these models are limited due to the perinatal lethality of GlcCerase gene. Here, we examined phenotypes of Drosophila melanogaster homologues genes of the human Gaucher's disease gene by using Minos insertion. One of two Minos insertion mutants to unknown function gene (CG31414) accumulates the hydroxy-GlcCer in whole body of Drosophila melanogaster. This mutant showed abnormal phenotypes of climbing ability and sleep, and short lifespan. These abnormal phenotypes are very similar to that of Gaucher's disease in human. In contrast, another Minos insertion mutant (CG31148) and its RNAi line did not show such severe phenotype as observed in CG31414 gene mutation. The data suggests that Drosophila CG31414 gene mutation might be useful for unraveling the molecular mechanism of Gaucher's disease.
人类戈谢病被认为是由于葡糖脑苷脂酶(GlcCerase)缺乏,导致其底物葡糖脑苷脂(GlcCer)蓄积。尽管多个实验室已报道了戈谢病的小鼠模型,但由于GlcCerase基因的围产期致死性,这些模型存在局限性。在此,我们通过使用Minos插入技术,研究了人类戈谢病基因在果蝇中的同源基因的表型。两个插入到功能未知基因(CG31414)的Minos插入突变体之一,在果蝇全身蓄积了羟基 - GlcCer。该突变体表现出攀爬能力和睡眠的异常表型,以及寿命缩短。这些异常表型与人类戈谢病的表型非常相似。相比之下,另一个Minos插入突变体(CG31148)及其RNA干扰品系并未表现出如CG31414基因突变所观察到的严重表型。数据表明,果蝇CG31414基因突变可能有助于阐明戈谢病的分子机制。