ALTERNATIVE DIETARY THERAPIES FOR GLYCOGEN STORAGE DISEASE TYPE 1

1 型糖原储存疾病的替代饮食疗法

• 批准号: 7717113
• 负责人: David Daniel Weinstein
• 金额: $ 2.06万
• 依托单位: UNIVERSITY OF FLORIDA
• 依托单位国家: 美国
• 财政年份: 2007
• 资助国家: 美国
• 起止时间: 2007-12-01 至 2008-11-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7717113
• 关键词: Computer Retrieval of Information on Scientific Projects Database; Corn starch preparation; Dose; Enzymes; Funding; Future; Gluconeogenesis; Glucose; Glycemic Index; Glycogen; Glycogen Storage Disease; Glycogen Storage Disease Type I; Goals; Grant; Hepatic; Hereditary Disease; Hypoglycemia; Institution; Kidney; Liver; Patients; Phenotype; Pilot Projects; Postabsorptive Hypoglycemia; Property; Purpose; Research; Research Personnel; Resources; Safety; Source; Starch; United States National Institutes of Health; absorption; blood glucose regulation; day; design; glucose-6-phosphatase; glycogenolysis

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.

该子项目是利用该技术的众多研究子项目之一 资源由 NIH/NCRR 资助的中心拨款提供。子项目和 研究者 (PI) 可能已从 NIH 的另一个来源获得主要资金， 因此可以在其他 CRISP 条目中表示。列出的机构是 对于中心来说，它不一定是研究者的机构。 糖原贮积病（GSD）是一组遗传性疾病，其表型为严重低血糖，因为肝脏和肾脏无法从糖原产生葡萄糖1-3。 I 型糖原贮积病 (GSDI) 是由肝葡萄糖 6 磷酸酶缺乏引起的，该酶是糖原分解和糖异生的重要组成部分 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。 目前患者平均每天接受 6.2 剂量的玉米淀粉治疗。 需要一种可消化的淀粉来为 GSD 患者提供餐后较长时间的葡萄糖来源。 这项试点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，其具有更好的血糖指数和延长葡萄糖吸收的能力。 这项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其重要性，而是为了确定未来研究的最佳药物。