Molecular Therapies for Cystic Fibrosis Lung Disease

囊性纤维化肺病的分子疗法

基本信息

  • 批准号:
    10470331
  • 负责人:
  • 金额:
    $ 231.16万
  • 依托单位:
  • 依托单位国家:
    美国
  • 项目类别:
  • 财政年份:
    2020
  • 资助国家:
    美国
  • 起止时间:
    2020-09-01 至 2025-07-31
  • 项目状态:
    未结题

项目摘要

OVERALL COMPONENT PROJECT SUMMARY Cystic fibrosis (CF) is a common life-shortening genetic disease that causes progressive lung failure due to recurrent infections and airway obstruction. While our knowledge of CFTR function has advanced greatly in the 30 years since the discovery of the gene, treatments for the disease remain suboptimal and CF remains progressive and fatal. Advances with small molecule CFTR modulator therapies have helped restore protein function for many mutations, but approximately 10% of people with CF have not benefited from these strategies, including people with nonsense and splicing mutations. The central theme of this proposal is developing new molecular therapies to prevent or treat CF lung disease. The goal of our three projects and four cores is to exploit the power of our in vitro and animal models to address questions fundamental to lung disease pathogenesis and to use this knowledge to inform new therapeutic strategies to complement CF defects, including gene repair and the addition of a small molecule that forms anion channels. The three closely interrelated Projects will work together to accomplish the following goals: 1) To restore CFTR function using targeted single nucleotide editing. We hypothesize that cells in the surface airway epithelium, including those with progenitor capacity, can be targeted to repair CFTR mutations using base editing. 2) To understand the mechanisms of amphotericin B (AmB)-induced anion secretion in airway epithelia and to test the hypothesis that AmB can restore CF host defenses in vivo. AmB is a small molecule that forms anion channels. 3) To determine how CFTR expression in pulmonary ionocytes and ciliated cells regulates properties of the airway surface liquid that are crucial for clearance and innate immunity. The development of effective gene therapies for cystic fibrosis lung disease must be guided by a clear understanding of pathophysiologic mechanisms of disease and the relevant cellular targets for CFTR gene replacement or editing. The Project Leaders and their teams have outstanding track records of collaborative CF research, and here they sharpen their focus to a common goal. Their highly creative research is supported by four cores that provide innovative infrastructure and services. Through these studies we hope to accelerate the development of new therapeutics for CF lung disease. !
整体组件

项目成果

期刊论文数量(0)
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会议论文数量(0)
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PAUL B MCCRAY其他文献

PAUL B MCCRAY的其他文献

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{{ truncateString('PAUL B MCCRAY', 18)}}的其他基金

Iowa StARR Scholars Program
爱荷华州 StarR 学者计划
  • 批准号:
    10565958
  • 财政年份:
    2021
  • 资助金额:
    $ 231.16万
  • 项目类别:
Iowa StARR Scholars Program
爱荷华州 StarR 学者计划
  • 批准号:
    10318208
  • 财政年份:
    2021
  • 资助金额:
    $ 231.16万
  • 项目类别:
Molecular Therapies for Cystic Fibrosis Lung Disease
囊性纤维化肺病的分子疗法
  • 批准号:
    10677580
  • 财政年份:
    2020
  • 资助金额:
    $ 231.16万
  • 项目类别:
Administration Core
行政核心
  • 批准号:
    10677581
  • 财政年份:
    2020
  • 资助金额:
    $ 231.16万
  • 项目类别:
Gene Editing Strategies to Correct CFTR Mutations
纠正 CFTR 突变的基因编辑策略
  • 批准号:
    10024666
  • 财政年份:
    2020
  • 资助金额:
    $ 231.16万
  • 项目类别:
Administration Core
行政核心
  • 批准号:
    10470332
  • 财政年份:
    2020
  • 资助金额:
    $ 231.16万
  • 项目类别:
Gene Editing Strategies to Correct CFTR Mutations
纠正 CFTR 突变的基因编辑策略
  • 批准号:
    10677600
  • 财政年份:
    2020
  • 资助金额:
    $ 231.16万
  • 项目类别:
Molecular Therapies for Cystic Fibrosis Lung Disease
囊性纤维化肺病的分子疗法
  • 批准号:
    10024661
  • 财政年份:
    2020
  • 资助金额:
    $ 231.16万
  • 项目类别:
Administration Core
行政核心
  • 批准号:
    10024662
  • 财政年份:
    2020
  • 资助金额:
    $ 231.16万
  • 项目类别:
Administration Core
行政核心
  • 批准号:
    10248524
  • 财政年份:
    2020
  • 资助金额:
    $ 231.16万
  • 项目类别:

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  • 批准号:
    10534031
  • 财政年份:
    2022
  • 资助金额:
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  • 项目类别:
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使用 FRET 开发 miRNA 和腺嘌呤甲基转移酶的诺贝尔检测方法
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    21K05120
  • 财政年份:
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  • 财政年份:
    2020
  • 资助金额:
    $ 231.16万
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胎盘滋养层发育中 N6-腺嘌呤 DNA 甲基化
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  • 财政年份:
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胎盘滋养层发育中 N6-腺嘌呤 DNA 甲基化
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