Department of Biology, Keio University, Yokohama, Japan.
Genes Cells. 2021 Jan;26(1):31-39. doi: 10.1111/gtc.12818. Epub 2020 Nov 28.
The periodic albino mutant of Xenopus laevis is a recessive mutant, in which reduced amounts of melanin appear in the retinal pigment epithelium (RPE) and in melanophores at the late embryonic stage, after which both RPE and melanophores gradually depigment. Three types of pigment cells (melanophores, iridophores and xanthophores) have been reported to be affected in this albino. However, the causative gene of the periodic albinism remains unknown. Hermansky-Pudlak syndrome (HPS) is an autosomal recessive disorder that affects humans and mice, which is caused by defective biogenesis of lysosome-related organelles (LROs). Two subgenomes (L and S) are present in the allotetraploid frog X. laevis. Comparison of genes between the chromosomes 1L and 1S revealed that the HPS type 4 (hps4) gene was present only in chromosome 1L. In the albino mutant, a 1.9 kb genomic deletion in the hps4.L gene including exons 7 and 8 caused a premature stop codon to create a truncated Hps4 protein. Injection of wild-type hps4.L mRNA into mutant embryos rescued the albino phenotype. These findings indicate that hps4 is a causative gene for the periodic albinism in X. laevis. The phenotype of this mutant should be reassessed from the perspective of LRO biogenesis.
非洲爪蟾的周期性白化突变体是一种隐性突变体,在胚胎后期,视网膜色素上皮(RPE)和黑素细胞中的黑色素含量减少,此后 RPE 和黑素细胞逐渐退色。在这种白化突变体中,已经报道有三种类型的色素细胞(黑素细胞、虹膜细胞和黄色素细胞)受到影响。然而,周期性白化病的致病基因仍然未知。Hermansky-Pudlak 综合征(HPS)是一种影响人类和小鼠的常染色体隐性疾病,是由溶酶体相关细胞器(LRO)生物发生缺陷引起的。四倍体青蛙 X. laevis 有两个亚基因组(L 和 S)。比较染色体 1L 和 1S 之间的基因发现,HPS 类型 4(hps4)基因仅存在于染色体 1L 上。在白化突变体中,hps4.L 基因的 1.9 kb 基因组缺失包括外显子 7 和 8,导致提前出现终止密码子,从而产生截断的 Hps4 蛋白。将野生型 hps4.L mRNA 注射到突变体胚胎中可挽救白化表型。这些发现表明 hps4 是 X. laevis 周期性白化病的致病基因。从 LRO 生物发生的角度重新评估该突变体的表型。