Development of a Urine Test for At-Home Monitoring of Blood Phe Levels for PKU

开发用于在家监测 PKU 血液 Phe 水平的尿液检测

基本信息

  • 批准号:
    10822515
  • 负责人:
  • 金额:
    $ 29.57万
  • 依托单位:
  • 依托单位国家:
    美国
  • 项目类别:
  • 财政年份:
    2023
  • 资助国家:
    美国
  • 起止时间:
    2023-09-20 至 2024-08-31
  • 项目状态:
    已结题

项目摘要

PROJECT SUMMARY/ABSTRACT This project proposes the development of technologies to provide low-cost monitoring of phenylalanine (Phe) levels in the blood by a simple, noninvasive, at-home urine test, in order to enable daily monitoring by individuals with phenylketonuria (PKU). Also known as phenylalanine hydroxylase deficiency, PKU is a rare inborn error of metabolism that occurs in the US at a frequency of about 1 in 15,000 children. If untreated, Phe levels in the blood stream rise to neurotoxic levels, leading to neurological damage and intellectual disability within a few months of birth. Following detection by newborn screening, treatment involves lifelong strict metabolic control using a low-Phe diet and medications to maintain blood Phe levels within a healthy range. If properly controlled, normal health and development can be expected. Dried blood spot samples allow monitoring of Phe levels, which are recommended weekly for the first five years of life and monthly thereafter. However, blood tests for Phe typically require 5 to 10 days for lab results to be returned, thus increasing susceptibility to unrecognized spikes in blood Phe levels. In recognition of this present problem, the National PKU Alliance has identified the development of an at-home test for daily Phe monitoring as one of the most pressing needs for the PKU community. In the same way that at-home glucose meters have enabled diabetics to engage in the daily monitoring and control of their condition, an at-home meter for PKU would enable critical control of Phe levels with the precision necessary to improve health and long-term outcomes. Members of our team at Clemson University have recently patented technologies that enable improved sensitivity for the optical readout of levels of a specific Phe metabolite in urine using a simple colorimetric method. This proposed project will expand upon these achievements to produce a complete, simple, inexpensive at-home urine test system, called the Rally Phe-nometer, to enable PKU patients to monitor their blood Phe levels on a daily basis in a noninvasive manner. Absorbent coupons with optimized color-sensitivity will be integrated on a complete disposable test that can be dipped into a urine sample and inserted into a low-cost meter device. This meter will be developed by Circa Bioscience to acquire an image of the urine test coupons, perform the necessary colorimetry measurements, and report the estimated blood Phe level to the user or caregiver. Analytical performance of the system will be validated in Aim 1. We will also partner with the Greenwood Genetic Center, the primary clinical facility in South Carolina for individuals with PKU, to demonstrate the correlation between metabolite levels present in urine and blood Phe levels and to validate the clinical basis for the Rally Phe- nometer. In addition to PKU, our innovative coupon design, combined with the increased sensitivity of the colorimetry readout, will enable the development of at-home urine tests for monitoring a wide variety of conditions, including other inborn errors of metabolism and kidney disease.
项目总结/文摘

项目成果

期刊论文数量(0)
专著数量(0)
科研奖励数量(0)
会议论文数量(0)
专利数量(0)

数据更新时间:{{ journalArticles.updateTime }}

{{ item.title }}
{{ item.translation_title }}
  • DOI:
    {{ item.doi }}
  • 发表时间:
    {{ item.publish_year }}
  • 期刊:
  • 影响因子:
    {{ item.factor }}
  • 作者:
    {{ item.authors }}
  • 通讯作者:
    {{ item.author }}

数据更新时间:{{ journalArticles.updateTime }}

{{ item.title }}
  • 作者:
    {{ item.author }}

数据更新时间:{{ monograph.updateTime }}

{{ item.title }}
  • 作者:
    {{ item.author }}

数据更新时间:{{ sciAawards.updateTime }}

{{ item.title }}
  • 作者:
    {{ item.author }}

数据更新时间:{{ conferencePapers.updateTime }}

{{ item.title }}
  • 作者:
    {{ item.author }}

数据更新时间:{{ patent.updateTime }}

Kevin D Champaigne其他文献

Kevin D Champaigne的其他文献

{{ item.title }}
{{ item.translation_title }}
  • DOI:
    {{ item.doi }}
  • 发表时间:
    {{ item.publish_year }}
  • 期刊:
  • 影响因子:
    {{ item.factor }}
  • 作者:
    {{ item.authors }}
  • 通讯作者:
    {{ item.author }}

{{ truncateString('Kevin D Champaigne', 18)}}的其他基金

Development of an Autism Spectrum Disorder Screening Test based upon Metabolic Profiling of Fresh Blood Samples
基于新鲜血液样本代谢分析的自闭症谱系障碍筛查测试的开发
  • 批准号:
    9466602
  • 财政年份:
    2018
  • 资助金额:
    $ 29.57万
  • 项目类别:

相似国自然基金

具有抗癌活性的天然产物金霉酸(Aureolic acids)全合成与选择性构建2-脱氧糖苷键
  • 批准号:
    22007039
  • 批准年份:
    2020
  • 资助金额:
    24.0 万元
  • 项目类别:
    青年科学基金项目
海洋放线菌来源聚酮类化合物Pteridic acids生物合成机制研究
  • 批准号:
  • 批准年份:
    2019
  • 资助金额:
    10.0 万元
  • 项目类别:
    省市级项目
手性Lewis Acids催化的分子内串联1,5-氢迁移/环合反应及其在构建结构多样性手性含氮杂环化合物中的应用
  • 批准号:
    21372217
  • 批准年份:
    2013
  • 资助金额:
    80.0 万元
  • 项目类别:
    面上项目
对空气稳定的新型的有机金属Lewis Acids催化剂制备、表征与应用研究
  • 批准号:
    21172061
  • 批准年份:
    2011
  • 资助金额:
    30.0 万元
  • 项目类别:
    面上项目
钛及含钛Lewis acids促臭氧/过氧化氢体系氧化性能的广普性、高效性及其机制
  • 批准号:
    21176225
  • 批准年份:
    2011
  • 资助金额:
    60.0 万元
  • 项目类别:
    面上项目
基于Zip Nucleic Acids引物对高度降解和低拷贝DNA检材的STR分型研究
  • 批准号:
    81072511
  • 批准年份:
    2010
  • 资助金额:
    31.0 万元
  • 项目类别:
    面上项目
海洋天然产物Makaluvic acids 的全合成及其对南海鱼虱存活的影响
  • 批准号:
    30660215
  • 批准年份:
    2006
  • 资助金额:
    21.0 万元
  • 项目类别:
    地区科学基金项目

相似海外基金

Lipid nanoparticle-mediated Inhalation delivery of anti-viral nucleic acids
脂质纳米颗粒介导的抗病毒核酸的吸入递送
  • 批准号:
    502577
  • 财政年份:
    2024
  • 资助金额:
    $ 29.57万
  • 项目类别:
CAREER: Highly Rapid and Sensitive Nanomechanoelectrical Detection of Nucleic Acids
职业:高度快速、灵敏的核酸纳米机电检测
  • 批准号:
    2338857
  • 财政年份:
    2024
  • 资助金额:
    $ 29.57万
  • 项目类别:
    Continuing Grant
Double Incorporation of Non-Canonical Amino Acids in an Animal and its Application for Precise and Independent Optical Control of Two Target Genes
动物体内非规范氨基酸的双重掺入及其在两个靶基因精确独立光学控制中的应用
  • 批准号:
    BB/Y006380/1
  • 财政年份:
    2024
  • 资助金额:
    $ 29.57万
  • 项目类别:
    Research Grant
Quantifying L-amino acids in Ryugu to constrain the source of L-amino acids in life on Earth
量化 Ryugu 中的 L-氨基酸以限制地球生命中 L-氨基酸的来源
  • 批准号:
    24K17112
  • 财政年份:
    2024
  • 资助金额:
    $ 29.57万
  • 项目类别:
    Grant-in-Aid for Early-Career Scientists
Synthetic analogues based on metabolites of omega-3 fatty acids protect mitochondria in aging hearts
基于 omega-3 脂肪酸代谢物的合成类似物可保护衰老心脏中的线粒体
  • 批准号:
    477891
  • 财政年份:
    2023
  • 资助金额:
    $ 29.57万
  • 项目类别:
    Operating Grants
Metabolomic profiles of responders and non-responders to an omega-3 fatty acids supplementation.
对 omega-3 脂肪酸补充剂有反应和无反应者的代谢组学特征。
  • 批准号:
    495594
  • 财政年份:
    2023
  • 资助金额:
    $ 29.57万
  • 项目类别:
Molecular recognition and enantioselective reaction of amino acids
氨基酸的分子识别和对映选择性反应
  • 批准号:
    23K04668
  • 财政年份:
    2023
  • 资助金额:
    $ 29.57万
  • 项目类别:
    Grant-in-Aid for Scientific Research (C)
Integrated understanding and manipulation of hypoxic cellular functions by artificial nucleic acids with hypoxia-accumulating properties
具有缺氧累积特性的人工核酸对缺氧细胞功能的综合理解和操纵
  • 批准号:
    23H02086
  • 财政年份:
    2023
  • 资助金额:
    $ 29.57万
  • 项目类别:
    Grant-in-Aid for Scientific Research (B)
Basic research toward therapeutic strategies for stress-induced chronic pain with non-natural amino acids
非天然氨基酸治疗应激性慢性疼痛策略的基础研究
  • 批准号:
    23K06918
  • 财政年份:
    2023
  • 资助金额:
    $ 29.57万
  • 项目类别:
    Grant-in-Aid for Scientific Research (C)
Molecular mechanisms how arrestins that modulate localization of glucose transporters are phosphorylated in response to amino acids
调节葡萄糖转运蛋白定位的抑制蛋白如何响应氨基酸而被磷酸化的分子机制
  • 批准号:
    23K05758
  • 财政年份:
    2023
  • 资助金额:
    $ 29.57万
  • 项目类别:
    Grant-in-Aid for Scientific Research (C)
{{ showInfoDetail.title }}

作者:{{ showInfoDetail.author }}

知道了