Cellular model for personalized CFTR-directed therapeutics.

用于个性化 CFTR 导向治疗的细胞模型。

基本信息

  • 批准号:
    9384797
  • 负责人:
  • 金额:
    $ 79.81万
  • 依托单位:
  • 依托单位国家:
    美国
  • 项目类别:
  • 财政年份:
    2017
  • 资助国家:
    美国
  • 起止时间:
    2017-08-01 至 2019-07-31
  • 项目状态:
    已结题

项目摘要

Project Abstract/Summary This proposal is a NHLBI-RFA (R44) to develop and validate a novel human cell-based diagnostic tool for predicting individual cystic fibrosis (CF) patient responses to drugs that improve the function of mutated forms of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. Large- scale in vitro expansion of human nasal epithelial cells is now feasible. The primary cells, when differentiated as air/liquid interface cultures (ALI), recapitulate a patient-specific CFTR phenotype and provide a sensitive and reproducible platform for testing CFTR-directed therapeutics. Transepithelial ion transport is a direct measure of CFTR function and in vitro demonstration of improved function of mutated CFTR has been shown to predict clinical benefit in specific patients. Nasal epithelial cells are collected from CF subjects, cultured, and cryopreserved to generate a bank of cells of known CFTR genotype. Cells are expanded, seeded onto filter supports for ALI culture (up to ~5,000 cultures/subject), differentiated, tested for CFTR expression and activity. The immediate goals of this project are to use the primary HNE cell cultures to: 1) establish reproducibility of inter-patient CFTR- drug response variability, 2) determine if in vitro responses predict long-term in vivo clinical benefit, and 3) identify rare mutations that respond to existing CFTR modulators. The long term goal is to show that this in vitro human cell model predicts individual patient responses to CFTR-directed therapeutics and to develop a diagnostic platform to guide optimization of personalized therapies for all CF patients.
项目摘要/总结

项目成果

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CALVIN U COTTON其他文献

CALVIN U COTTON的其他文献

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{{ truncateString('CALVIN U COTTON', 18)}}的其他基金

ALTERED COLLECTING TUBULE ION TRANSPORT IN ARPKD
ARPKD 中收集管离子传输的改变
  • 批准号:
    6655217
  • 财政年份:
    2002
  • 资助金额:
    $ 79.81万
  • 项目类别:
ALTERED COLLECTING TUBULE ION TRANSPORT IN ARPKD
ARPKD 中收集管离子传输的改变
  • 批准号:
    6493082
  • 财政年份:
    2001
  • 资助金额:
    $ 79.81万
  • 项目类别:
SALT AND WATER TRANSPORT IN SMALL PANCREATIC DUCTS
小胰管中盐和水的运输
  • 批准号:
    6301073
  • 财政年份:
    2000
  • 资助金额:
    $ 79.81万
  • 项目类别:
REGULATION OF CHLORIDE ION CONDUCTANCE IN PANCREAS DUCT
胰管内氯离子电导的调节
  • 批准号:
    2855308
  • 财政年份:
    1999
  • 资助金额:
    $ 79.81万
  • 项目类别:
ALTERED COLLECTING TUBULE ION TRANSPORT IN ARPKD
ARPKD 中收集管离子传输的改变
  • 批准号:
    6195015
  • 财政年份:
    1999
  • 资助金额:
    $ 79.81万
  • 项目类别:
SALT AND WATER TRANSPORT IN SMALL PANCREATIC DUCTS
小胰管中盐和水的运输
  • 批准号:
    6201837
  • 财政年份:
    1999
  • 资助金额:
    $ 79.81万
  • 项目类别:
REGULATION OF CHLORIDE ION CONDUCTANCE IN PANCREAS DUCT
胰管内氯离子电导的调节
  • 批准号:
    6178078
  • 财政年份:
    1999
  • 资助金额:
    $ 79.81万
  • 项目类别:
REGULATION OF CHLORIDE ION CONDUCTANCE IN PANCREAS DUCT
胰管内氯离子电导的调节
  • 批准号:
    6517434
  • 财政年份:
    1999
  • 资助金额:
    $ 79.81万
  • 项目类别:
REGULATION OF CHLORIDE ION CONDUCTANCE IN PANCREAS DUCT
胰管内氯离子电导的调节
  • 批准号:
    6381061
  • 财政年份:
    1999
  • 资助金额:
    $ 79.81万
  • 项目类别:
SALT AND WATER TRANSPORT IN SMALL PANCREATIC DUCTS
小胰管中盐和水的运输
  • 批准号:
    6105189
  • 财政年份:
    1998
  • 资助金额:
    $ 79.81万
  • 项目类别:

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物理和生物模型的非局部变分问题
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