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Mitochondrial Genome Manipulation in Human Neuroepithelial Precursor Cells

人神经上皮前体细胞的线粒体基因组操作

基本信息

批准号：
7333972
负责人：
JAMES PEPPER BENNETT
金额：
$ 19.01万
依托单位：
UNIVERSITY OF VIRGINIA
依托单位国家：
美国
项目类别：
财政年份：
2007
资助国家：
美国
起止时间：
2007-07-01 至 2008-06-30
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/7333972
关键词：
Adult Alzheimer&apos s Disease American Amyloid beta-Protein Precursor Animal Model Animals Apoptotic Astrocytes Award Beds Biomedical Engineering Brain Care given by nurses Cell Death Cell Line Cell Lineage Cell model Cells Cellular biology Chemicals Communities Cost of Illness DNA Development Disasters Disease Disease model Doctor of Medicine Doctor of Philosophy Economics Educational workshop Faculty Failure Future Genes Goals Grant Healthcare Systems Human Human Cell Line In Vitro Incentives Individual Industry Learning Left Logic Medical Methods Mitochondria Mitochondrial DNA Modeling Mus Mutation Nerve Degeneration Neurodegenerative Disorders Neuroepithelial Neuroepithelial, Perineurial, and Schwann Cell Neoplasm Neuroglia Neurology Neurons Neuroprotective Agents Numbers Oligodendroglia Output Parkinson Disease Pathogenesis Pathology Patients Pharmaceutical Preparations Phenotype Physicians Play Preclinical Drug Evaluation Presenile Alzheimer Dementia Process Reporting Research Research Personnel Role Rotenone Science Scientist Small Business Technology Transfer Research Source Standards of Weights and Measures Study models TNFRSF5 gene Techniques Technology Transfer Testing Therapeutic Transgenic Model Transgenic Organisms United States National Institutes of Health Universities Virginia Vulnerable Populations Work Writing alpha synuclein base brain cell career cost dopaminergic neuron drug development familial Alzheimer disease gene therapy human disease human embryonic stem cell human embryonic stem cell line immunoglobulin deposition disease improved in vivo insight member mitochondrial genome neoplastic neoplastic cell nerve stem cell neurotoxic novel parkin gene/protein pre-clinical precursor cell presenilin-1 prevent professor programs relating to nervous system respite care response selective expression socioeconomics success tau Proteins ubiquitin-protein ligase

项目摘要

DESCRIPTION (provided by applicant): This is an application filed in response to RFA-DC-07-005 "Short-Term Interdisciplinary Career Enhancement Awards for Neuro-degeneration Research". The proposed candidate is James P. Bennett, Jr. M.D., Ph.D., who is currently Arthur and Margaret Ebbert Professor of Medical Science and Professor of Neurology at the University of Virginia (UVA). Dr. Bennett's entire 23-year academic career as a translational physician-scientist faculty member has been devoted to neurodegenerative research. He has held multiple NIH awards, beginning with a RCDA from 1985-1990, with subsequent awards of four R01 grants, two Program Projects and the P50 Udall Parkinson's Center at UVA that recently was competitively renewed through 2010. He has had significant translational output and presently holds a Physician-Sponsor IND for a novel neuroprotective drug and is PI for a STTR grant to develop mitochondrial gene therapy. He is a nationally known investigator into the mitochondrial pathobiology of neurodegenerative diseases. In the proposed career enhancement plan Dr. Bennett will learn how to derive neuroepithelial precursor (NEP) cells from NIH-approved human embryonic stem cells (hESC). He will learn this technique by attending a NIH-sponsored workshop followed by working closely with Dr. Raj Rao, a bioengineer on the faculty at Virginia Commonwealth University (VCU) and expert in neural stem cell biology. The goal of this project is for Dr. Bennett to create novel cell lines to serve as improved models of Alzheimer's (AD) and Parkinson's (PD) diseases by using a novel mitochondrial DNA (mtDNA) transfer technology to transfer AD and PD mtDNA into NEP cells depleted of their own mtDNA. These novel cell lines will allow exploration of unique disease pathogenesis hypotheses and will also serve as excellent drug screening platforms. The Award will allow Dr. Bennett to devote a 65% effort to this goal over 12 months of support. The precise causes of premature nerve cell death in Alzheimer's and Parkinson's patients' brains remain unknown. This award will support the creation of novel human nerve cells that carry mitochondrial genes from Alzheimer's and Parkinson's patients. These nerve cell lines can help scientists understand why nerve cells die prematurely in these diseases and can be used to develop new drugs to prevent cell death.

描述（由申请人提供）：这是响应RFA-DC-07-005“神经变性研究短期跨学科职业提升奖”的申请。提名的候选人是小詹姆斯·P·班尼特。医学博士，哲学博士、他目前是弗吉尼亚大学（UVA）医学科学的亚瑟和玛格丽特埃伯特教授和神经学教授。班尼特博士作为一名翻译医学家和科学家的整个23年的学术生涯一直致力于神经退行性疾病的研究。他曾获得多项NIH奖项，从1985年至1990年的RCDA开始，随后获得了四项R 01赠款，两项计划项目和UVA的P50 Udall帕金森中心，该中心最近在2010年进行了竞争性更新。他有显著的翻译输出，目前持有一种新型神经保护药物的医生申办者IND，并且是STTR资助开发线粒体基因疗法的PI。他是全国知名的神经退行性疾病的线粒体病理学研究者。在拟议的职业发展计划中，班尼特博士将学习如何从NIH批准的人类胚胎干细胞（hESC）中获得神经上皮前体（NEP）细胞。他将通过参加NIH赞助的研讨会来学习这项技术，然后与弗吉尼亚联邦大学（VCU）的生物工程师兼神经干细胞生物学专家Raj Rao博士密切合作。该项目的目标是为班尼特博士创建新的细胞系，作为阿尔茨海默氏症（AD）和帕金森氏症（PD）疾病的改进模型，通过使用一种新的线粒体DNA（mtDNA）转移技术，将AD和PD mtDNA转移到NEP细胞耗尽自己的mtDNA。这些新的细胞系将允许探索独特的疾病发病机制假设，也将作为优秀的药物筛选平台。该奖项将允许班尼特博士在12个月的支持中为这一目标付出65%的努力。阿尔茨海默氏症和帕金森氏症患者大脑中神经细胞过早死亡的确切原因仍然未知。该奖项将支持创造携带阿尔茨海默氏症和帕金森氏症患者线粒体基因的新型人类神经细胞。这些神经细胞系可以帮助科学家了解为什么神经细胞在这些疾病中过早死亡，并可用于开发预防细胞死亡的新药。