ALTERNATIVE DIETARY THERAPIES FOR GLYCOGEN STORAGE DISEASE TYPE 1

这个子项目是许多研究子项目中的一个 由NIH/NCRR资助的中心赠款提供的资源。子项目及 研究者（PI）可能从另一个NIH来源获得了主要资金， 因此可以在其他CRISP条目中表示。所列机构为 研究中心，而研究中心不一定是研究者所在的机构。 糖原累积病（GSD）是一组遗传性疾病，其中表型是严重的低血糖症，因为不能从肝脏和肾脏中的糖原产生葡萄糖1-3。 I型糖原累积病（GSDI）是由肝葡萄糖-6-磷酸酶缺乏引起的，而肝葡萄糖-6-磷酸酶是糖原分解和糖原生成的组成部分3。 缺乏这种酶会导致无法维持正常的葡萄糖稳态和严重的空腹低血糖。 目前，患者平均每天用6.2剂玉米淀粉治疗。 需要一种可消化的淀粉，其可以在GSD患者的餐后提供更长时间的葡萄糖来源。 这项初步研究的目的是探索一种改性玉米淀粉，与用于这些患者的传统玉米淀粉疗法相比，其特性表明血糖指数更好，并且能够提供延长葡萄糖吸收的能力。 本研究的主要目标是葡萄糖来源的耐受性和安全性。 这项研究的目的不是为了证明意义，而是为了确定未来研究的最佳药物。

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. The glycogen storage diseases (GSD) are a set of genetic disorders in which the phenotype is severe hypoglycemia because of the inability to produce glucose from glycogen in the liver and kidney 1-3. Type I glycogen storage disease (GSDI) is caused by a deficiency of hepatic glucose-6-phosphatase, which is an integral part of both glycogenolysis and gluconeogenesis 3. Lack of this enzyme results in the inability to maintain normal glucose homeostasis and severe fasting hypoglycemia. Patients presently are treated with an average of 6.2 doses of cornstarch per day. There is a need for a digestible starch that can provide a source of glucose for longer duration after meals in GSD patients. The purpose of this pilot study is to explore a modified cornstarch with properties that suggest a better glycemic index and the ability to provide prolonged glucose absorption compared to the traditional cornstarch therapy used for these patients. The main goals of this study will be the tolerance and safety of the glucose sources. This study is not designed to demonstrate significance, but rather to identify the best agent for future studies.