Peng Wen-Tao, Pan Chi-Jiunn, Lee Eric J, Westphal Heiner, Chou Janice Y
Section on Cellular Differentiation, Program on Developmental Endocrinology and Genetics, National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, Maryland, USA.
Genesis. 2009 Sep;47(9):590-4. doi: 10.1002/dvg.20538.
Glucose-6-phosphatase-alpha (G6Pase-alpha or G6PC) catalyzes the hydrolysis of glucose-6-phosphate to glucose and is a key enzyme in interprandial glucose homeostasis. Mutations in the human G6PC gene, expressed primarily in the liver, kidney, and intestine, cause glycogen storage disease Type Ia (GSD-Ia), an autosomal recessive disorder characterized by a disturbed glucose homeostasis. For better understanding of the roles of G6Pase-alpha in different tissues and in pathological conditions, we have generated mice harboring a conditional null allele for G6pc by flanking Exon 3 of the G6pc gene with loxP sites. We confirmed the null phenotype by using the EIIa-Cre transgenic approach to generate mice lacking Exon 3 of the G6pc gene. The resulting homozygous Cre-recombined null mice manifest a phenotype mimicking G6Pase-alpha-deficient mice and human GSD-Ia patients. This G6pc conditional null allele will be valuable to examine the consequence of tissue-specific G6Pase-alpha deficiency and the mechanisms of long-term complications in GSD-Ia.
葡萄糖-6-磷酸酶-α(G6Pase-α或G6PC)催化葡萄糖-6-磷酸水解为葡萄糖,是餐间葡萄糖稳态中的关键酶。人G6PC基因主要在肝脏、肾脏和肠道中表达,该基因突变会导致I型糖原贮积病(GSD-Ia),这是一种常染色体隐性疾病,其特征为葡萄糖稳态紊乱。为了更好地了解G6Pase-α在不同组织和病理状况中的作用,我们通过在G6pc基因的外显子3两侧插入loxP位点,构建了携带G6pc条件性无效等位基因的小鼠。我们使用EIIa-Cre转基因方法产生缺失G6pc基因外显子3的小鼠,从而证实了无效表型。由此产生的纯合Cre重组无效小鼠表现出与G6Pase-α缺陷小鼠和人类GSD-Ia患者相似的表型。这种G6pc条件性无效等位基因对于研究组织特异性G6Pase-α缺陷的后果以及GSD-Ia长期并发症的机制将具有重要价值。