Core C

核心C

基本信息

  • 批准号:
    10582720
  • 负责人:
  • 金额:
    $ 20.35万
  • 依托单位:
  • 依托单位国家:
    美国
  • 项目类别:
  • 财政年份:
    2014
  • 资助国家:
    美国
  • 起止时间:
    2014-09-30 至 2025-03-31
  • 项目状态:
    未结题

项目摘要

PROJECT SUMMARY/ABSTRACT: CORE C G4C2 repeat expansions in C9orf72 are the most common genetic cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), two devastating diseases with no effective treatment. Success in developing a treatment for “c9ALS/FTD” will require a better understanding of disease pathomechanisms, the identification of molecular pathways amenable to therapeutic targeting, and the development of biomarkers to track disease progression and to confirm target engagement of potential therapies. The studies outlined in Projects 1, 2 and 3 aim to fill these needs, and the Human Validation Core (Core C) will work closely with Project investigators to facilitate these important studies. In addition to providing post-mortem tissues to Projects 1, 2 and 3, Core C will conduct a thorough neuropathological and biochemical analysis of these patient samples, including the quantification of TDP-43, P62 and dipeptide repeat (DPR) protein pathology, microgliosis and astrogliosis, and repeat length. Core C will similarly characterize brain tissues from (G4C2)149 mice used in Projects 1 and 3, and measure DPR protein levels in neurons differentiated from patient-derived induced pluripotent stem cells used in Project 2. These data will serve as valuable traits for the proteomic and transcriptomic studies conducted in all three projects to discover pathological mechanisms relevant to disease, and biomarkers for use in clinical trials. Furthermore, Core C will play a significant role in validating, at the protein level, expression changes of genes or proteins that associate with ALS genotypes or phenotypes, and that may define pathophysiological pathways linked to c9ALS. This will be done with independent methods of protein detection and using tissues from discovery series and validation series. Indeed, to ensure that the collection of post-mortem tissues from C9orf72 repeat expansion carriers continues to grow, Core C will work closely with Core B to obtain rapid autopsies of patients enrolled in clinical studies and provide diagnostic evaluations. In this fashion, we will further increase the number of patients from whom both antemortem cerebrospinal fluid (CSF) and postmortem brain tissue are available – important resources for comparing brain and CSF proteomes. With regards to the latter, Core C will help Project 3 home in on promising candidate biomarkers that warrant investigation in CSF, and assist in determining whether their top candidates could serve as CSF biomarkers for c9ALS/FTD.
项目摘要/摘要:核心c

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)

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Tania France Gendron其他文献

Tania France Gendron的其他文献

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{{ truncateString('Tania France Gendron', 18)}}的其他基金

Poly(ADP-ribose) promotes the condensation and toxicity of C9orf72 arginine-rich dipeptide repeat proteins
聚(ADP-核糖)促进 C9orf72 富含精氨酸的二肽重复蛋白的缩合和毒性
  • 批准号:
    10707931
  • 财政年份:
    2021
  • 资助金额:
    $ 20.35万
  • 项目类别:
Poly(ADP-ribose) promotes the condensation and toxicity of C9orf72 arginine-rich dipeptide repeat proteins
聚(ADP-核糖)促进 C9orf72 富含精氨酸的二肽重复蛋白的缩合和毒性
  • 批准号:
    10298581
  • 财政年份:
    2021
  • 资助金额:
    $ 20.35万
  • 项目类别:
Poly(ADP-ribose) promotes the condensation and toxicity of C9orf72 arginine-rich dipeptide repeat proteins
聚(ADP-核糖)促进 C9orf72 富含精氨酸的二肽重复蛋白的缩合和毒性
  • 批准号:
    10448297
  • 财政年份:
    2021
  • 资助金额:
    $ 20.35万
  • 项目类别:
Frontotemporal dementia and related disorders: transcriptomic profiling, biomarker discovery, and mechanistic insight
额颞叶痴呆及相关疾病:转录组分析、生物标志物发现和机制洞察
  • 批准号:
    10392505
  • 财政年份:
    2021
  • 资助金额:
    $ 20.35万
  • 项目类别:
Frontotemporal dementia and related disorders: transcriptomic profiling, biomarker discovery, and mechanistic insight
额颞叶痴呆及相关疾病:转录组分析、生物标志物发现和机制洞察
  • 批准号:
    10180652
  • 财政年份:
    2021
  • 资助金额:
    $ 20.35万
  • 项目类别:
Frontotemporal dementia and related disorders: transcriptomic profiling, biomarker discovery, and mechanistic insight
额颞叶痴呆及相关疾病:转录组分析、生物标志物发现和机制洞察
  • 批准号:
    10597677
  • 财政年份:
    2021
  • 资助金额:
    $ 20.35万
  • 项目类别:
Core C
核心C
  • 批准号:
    10415045
  • 财政年份:
    2014
  • 资助金额:
    $ 20.35万
  • 项目类别:
Pathologically modified TDP-43 in neurodegenerative diseases
病理修饰的 TDP-43 在神经退行性疾病中的应用
  • 批准号:
    8263381
  • 财政年份:
    2011
  • 资助金额:
    $ 20.35万
  • 项目类别:
Pathologically modified TDP-43 in neurodegenerative diseases
病理修饰的 TDP-43 在神经退行性疾病中的应用
  • 批准号:
    8093934
  • 财政年份:
    2011
  • 资助金额:
    $ 20.35万
  • 项目类别:

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