Microphysiological Human Tissue Systems for Monitoring of Genome Editing Outcomes

用于监测基因组编辑结果的微生理人体组织系统

基本信息

  • 批准号:
    9810917
  • 负责人:
  • 金额:
    $ 66.82万
  • 依托单位:
  • 依托单位国家:
    美国
  • 项目类别:
  • 财政年份:
    2019
  • 资助国家:
    美国
  • 起止时间:
    2019-09-01 至 2023-08-31
  • 项目状态:
    已结题

项目摘要

Abstract: Genome editing technologies have significant potential to treat a variety of devastating human diseases and disorders. However, there are a number of challenges that genome editing therapies must overcome to reach their full promise. Specifically, there are many possible adverse consequences that are unique to genome editing tools, such as genome integrity, immune responses, and loss of therapeutic efficacy due to cell turnover, for which there are currently are no optimal systems for rigorous assessment. Moreover, these consequences are unique to human physiology, genome sequence, and immune systems, and therefore typical animal models are not completely informative. To address this unmet need, we have assembled a team of collaborative investigators that have developed advanced genome editing strategies and methods for engineering human microphysiological tissue systems that recapitulate human physiology and function, with an emphasis on skeletal and cardiac muscle. We will combine these technologies in this proposal to systemically evaluate tissue physiology, genomic alterations, tissue regeneration, and immune response in response to various genome editing strategies and delivery methods. Specifically, this will include comprehensive and unbiased mapping of unintended modifications to human genome sequences, including at on-target and off-target sites. We will also determine the role of resident tissue stem cells, cell turnover, and tissue injury and regeneration in the stability of genome editing. Finally, we can incorporate immune cells into these microphysiological tissues to understand the consequences of immunity to bacteria-derived genome editing components. Collectively, this proposal will develop a platform to systematically address the most significant challenges to realizing the transformative potential of genome editing therapies in human tissues.
文摘:

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)

数据更新时间:{{ journalArticles.updateTime }}

{{ item.title }}
{{ item.translation_title }}
  • DOI:
    {{ item.doi }}
  • 发表时间:
    {{ item.publish_year }}
  • 期刊:
  • 影响因子:
    {{ item.factor }}
  • 作者:
    {{ item.authors }}
  • 通讯作者:
    {{ item.author }}

数据更新时间:{{ journalArticles.updateTime }}

{{ item.title }}
  • 作者:
    {{ item.author }}

数据更新时间:{{ monograph.updateTime }}

{{ item.title }}
  • 作者:
    {{ item.author }}

数据更新时间:{{ sciAawards.updateTime }}

{{ item.title }}
  • 作者:
    {{ item.author }}

数据更新时间:{{ conferencePapers.updateTime }}

{{ item.title }}
  • 作者:
    {{ item.author }}

数据更新时间:{{ patent.updateTime }}

Nenad Bursac其他文献

Nenad Bursac的其他文献

{{ item.title }}
{{ item.translation_title }}
  • DOI:
    {{ item.doi }}
  • 发表时间:
    {{ item.publish_year }}
  • 期刊:
  • 影响因子:
    {{ item.factor }}
  • 作者:
    {{ item.authors }}
  • 通讯作者:
    {{ item.author }}

{{ truncateString('Nenad Bursac', 18)}}的其他基金

Engineering a Human Skeletal Muscle Tissue Model of LGMD2B
设计 LGMD2B 的人体骨骼肌组织模型
  • 批准号:
    10719721
  • 财政年份:
    2023
  • 资助金额:
    $ 66.82万
  • 项目类别:
Engineering Human Heart Tissues with Polyploid Cardiomyocytes
用多倍体心肌细胞改造人类心脏组织
  • 批准号:
    10467794
  • 财政年份:
    2022
  • 资助金额:
    $ 66.82万
  • 项目类别:
Engineering Human Heart Tissues with Polyploid Cardiomyocytes
用多倍体心肌细胞改造人类心脏组织
  • 批准号:
    10616611
  • 财政年份:
    2022
  • 资助金额:
    $ 66.82万
  • 项目类别:
Engineered BacNav and BacCav for Improved Excitability and Contraction
专为改善兴奋性和收缩性而设计的 BacNav 和 BacCav
  • 批准号:
    10392121
  • 财政年份:
    2022
  • 资助金额:
    $ 66.82万
  • 项目类别:
Engineered BacNav and BacCav for Improved Excitability and Contraction
专为改善兴奋性和收缩性而设计的 BacNav 和 BacCav
  • 批准号:
    10611385
  • 财政年份:
    2022
  • 资助金额:
    $ 66.82万
  • 项目类别:
Microphysiological Human Tissue Systems for Monitoring of Genome Editing Outcomes
用于监测基因组编辑结果的微生理人体组织系统
  • 批准号:
    10001507
  • 财政年份:
    2019
  • 资助金额:
    $ 66.82万
  • 项目类别:
Microphysiological Human Tissue Systems for Monitoring of Genome Editing Outcomes
用于监测基因组编辑结果的微生理人体组织系统
  • 批准号:
    10242833
  • 财政年份:
    2019
  • 资助金额:
    $ 66.82万
  • 项目类别:
Microphysiological Human Tissue Systems for Monitoring of Genome Editing Outcomes
用于监测基因组编辑结果的微生理人体组织系统
  • 批准号:
    10477016
  • 财政年份:
    2019
  • 资助金额:
    $ 66.82万
  • 项目类别:
Engineering of Human Excitable Tissues from Unexcitable Cells
从不可兴奋细胞改造人类可兴奋组织
  • 批准号:
    9270588
  • 财政年份:
    2016
  • 资助金额:
    $ 66.82万
  • 项目类别:
Engineering of Human Excitable Tissues from Unexcitable Cells
从不可兴奋细胞改造人类可兴奋组织
  • 批准号:
    9046968
  • 财政年份:
    2016
  • 资助金额:
    $ 66.82万
  • 项目类别:

相似海外基金

How Does Particle Material Properties Insoluble and Partially Soluble Affect Sensory Perception Of Fat based Products
不溶性和部分可溶的颗粒材料特性如何影响脂肪基产品的感官知觉
  • 批准号:
    BB/Z514391/1
  • 财政年份:
    2024
  • 资助金额:
    $ 66.82万
  • 项目类别:
    Training Grant
BRC-BIO: Establishing Astrangia poculata as a study system to understand how multi-partner symbiotic interactions affect pathogen response in cnidarians
BRC-BIO:建立 Astrangia poculata 作为研究系统,以了解多伙伴共生相互作用如何影响刺胞动物的病原体反应
  • 批准号:
    2312555
  • 财政年份:
    2024
  • 资助金额:
    $ 66.82万
  • 项目类别:
    Standard Grant
RII Track-4:NSF: From the Ground Up to the Air Above Coastal Dunes: How Groundwater and Evaporation Affect the Mechanism of Wind Erosion
RII Track-4:NSF:从地面到沿海沙丘上方的空气:地下水和蒸发如何影响风蚀机制
  • 批准号:
    2327346
  • 财政年份:
    2024
  • 资助金额:
    $ 66.82万
  • 项目类别:
    Standard Grant
Graduating in Austerity: Do Welfare Cuts Affect the Career Path of University Students?
紧缩毕业:福利削减会影响大学生的职业道路吗?
  • 批准号:
    ES/Z502595/1
  • 财政年份:
    2024
  • 资助金额:
    $ 66.82万
  • 项目类别:
    Fellowship
感性個人差指標 Affect-X の構築とビスポークAIサービスの基盤確立
建立个人敏感度指数 Affect-X 并为定制人工智能服务奠定基础
  • 批准号:
    23K24936
  • 财政年份:
    2024
  • 资助金额:
    $ 66.82万
  • 项目类别:
    Grant-in-Aid for Scientific Research (B)
Insecure lives and the policy disconnect: How multiple insecurities affect Levelling Up and what joined-up policy can do to help
不安全的生活和政策脱节:多种不安全因素如何影响升级以及联合政策可以提供哪些帮助
  • 批准号:
    ES/Z000149/1
  • 财政年份:
    2024
  • 资助金额:
    $ 66.82万
  • 项目类别:
    Research Grant
How does metal binding affect the function of proteins targeted by a devastating pathogen of cereal crops?
金属结合如何影响谷类作物毁灭性病原体靶向的蛋白质的功能?
  • 批准号:
    2901648
  • 财政年份:
    2024
  • 资助金额:
    $ 66.82万
  • 项目类别:
    Studentship
Investigating how double-negative T cells affect anti-leukemic and GvHD-inducing activities of conventional T cells
研究双阴性 T 细胞如何影响传统 T 细胞的抗白血病和 GvHD 诱导活性
  • 批准号:
    488039
  • 财政年份:
    2023
  • 资助金额:
    $ 66.82万
  • 项目类别:
    Operating Grants
New Tendencies of French Film Theory: Representation, Body, Affect
法国电影理论新动向:再现、身体、情感
  • 批准号:
    23K00129
  • 财政年份:
    2023
  • 资助金额:
    $ 66.82万
  • 项目类别:
    Grant-in-Aid for Scientific Research (C)
The Protruding Void: Mystical Affect in Samuel Beckett's Prose
突出的虚空:塞缪尔·贝克特散文中的神秘影响
  • 批准号:
    2883985
  • 财政年份:
    2023
  • 资助金额:
    $ 66.82万
  • 项目类别:
    Studentship
{{ showInfoDetail.title }}

作者:{{ showInfoDetail.author }}

知道了