Kits to Quantitate and Analyze Mitochondrial Proteins

定量和分析线粒体蛋白的试剂盒

基本信息

批准号：
7053255
负责人：
Michael F Marusich
金额：
$ 37.5万
依托单位：
MITOSCIENCES, INC
依托单位国家：
美国
项目类别：
财政年份：
2004
资助国家：
美国
起止时间：
2004-06-07 至 2008-02-29
项目状态：
已结题

项目摘要

DESCRIPTION (provided by applicant): Mitochondrial dysfunction underlies the pathology of many disorders, including inherited mitochondrial diseases, many degenerative diseases, and the adverse effects of certain therapeutic drugs. At present there are no reliable standardized methods for detection and analysis of these defects. Our goal is to meet these needs with a set of novel assay kits that simplify analysis of key mitochondrial proteins and add powerful new analytical capabilities. These innovative kits are based on a unique set of proprietary reagents and associated intellectual properties developed by the Principal Investigators. The reagents are monoclonal antibodies (mAbs) that immunocapture intact, functional mitochondrial enzymes, and the intellectual properties are patents (pending) that cover these mAbs and their application. The Phase II STTR grant will drive commercialization of these technologies in three areas: 1) pharmaceutical drug safety screening for mitotoxicity, both to guide new drug development and to monitor adverse effects of licensed therapeutic drugs; 2) diagnosis of mitochondrial disorders (inherited and acquired) with simple standardized tests; and 3) research applications ranging from basic research into mitochondrial structure/function, to applied research that will help identify biomarkers (pathogenic and diagnostic) of mitochondrial disease. We have successfully met our Phase I goals and also accomplished additional work proactively. Working prototypes of all three types of kits are in hand and we have documented their basic performance characteristics. We are currently marketing early versions of some kits, and have established that there is a strong scientific and commercial demand for them. Phase II work will complete the development of these and other tests and validate their technical utility, making them ready for commercialization. Relevance in lay language: Mitochondria are/the powerhouse of cells and produce 95% of the energy needed for life. The proposed kits will enable personalized mitochondrial medicine, including measurement of an individual's mitotoxic burden (the degree to which his/her mitochondrial energy production system is damaged), and allow assessment of an individual's sensitivity to mitotoxic agents (including adverse effects of therapeutic drugs and environmental toxins). The kits will also be used to detect and monitor the progression of inherited mitochondrial diseases and degenerative diseases such as Parkinson's and Type-2 diabetes, and this will help clinicians determine whether or not new therapies for these disorders provide beneficial results. Finally, they will offer a way to for new therapeutic drugs that can protect mitochondria from toxic agents, and thus potentially prevent or treat mitochondrial disorders.

描述（由申请人提供）：线粒体功能障碍是许多疾病的病理基础，包括遗传性线粒体疾病，许多退化性疾病以及某些治疗药物的不良影响。目前，没有可靠的标准化方法来检测和分析这些缺陷。我们的目标是通过一套新颖的测定套件来满足这些需求，以简化对关键线粒体蛋白的分析并添加强大的新分析能力。这些创新的套件基于主要研究人员开发的一系列独特的专有试剂和相关的知识产权。试剂是单克隆抗体（mAb），即免疫接触完整，功能性线粒体酶和智力特性是涵盖这些mAb及其应用的专利（待处理）。 II期STTR赠款将在三个领域推动这些技术的商业化：1）药物安全筛查的丝毒性，以指导新药开发和监测有执照的治疗药物的不良影响； 2）用简单的标准化测试诊断线粒体疾病（遗传和获取）； 3）研究应用程序从线粒体结构/功能的基础研究到应用研究，这些研究将有助于鉴定线粒体疾病的生物标志物（致病性和诊断）。我们已经成功实现了I阶段目标，并积极完成了其他工作。所有三种套件的工作原型都在手中，我们已经记录了它们的基本性能特征。我们目前正在销售某些套件的早期版本，并确定对它们有强烈的科学和商业需求。第二阶段的工作将完成这些和其他测试的开发，并验证其技术公用事业，使其为商业化做好准备。与外行语言相关：线粒体是/是细胞的动力，产生了95％的生命能量。拟议的试剂盒将使个性化的线粒体医学能够测量个体的丝毒性负担（其线粒体能源生产系统的损害程度），并允许评估个人对有毒剂的敏感性（包括对治疗药物和环境毒素的不良影响）。这些套件还将用于检测和监测遗传性线粒体疾病和退行性疾病（例如帕金森氏症和2型糖尿病）的进展，这将有助于临床医生确定这些疾病的新疗法是否提供有益的结果。最后，他们将提供一种方法，用于可以保护线粒体免受有毒剂的新治疗药物，从而有可能预防或治疗线粒体疾病。