Mechanisms and optimization of endosomal escape for delivery applications

递送应用的内体逃逸机制和优化

• 批准号: 10388856
• 负责人: Jean-Philippe Pellois
• 金额: $ 3.27万
• 依托单位: TEXAS A&M AGRILIFE RESEARCH
• 依托单位国家: 美国
• 财政年份: 2015
• 资助国家: 美国
• 起止时间: 2015-06-01 至 2023-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10388856
• 关键词: Biological; CRISPR/Cas technology; Cell Culture Techniques; Cells; Cellular Membrane; Goals; Membrane; Nucleic Acids; Problem Solving; Proteins; Reagent; Techniques; Therapeutic Intervention; Work; cell type; human disease; in vivo; macromolecule; nucleic acid-based therapeutics; therapeutic development

PROJECT SUMMARY/ABSTRACT Title: Mechanisms and optimization of endosomal escape for cell delivery applications Controlled manipulation of cells through the precise intracellular delivery of biologically active materials has been a long-term goal for probing of cellular mechanisms and therapeutic interventions. Cellular delivery is however a problem that has not yet been solved. Most techniques remain inefficient, are disruptive to cells and can be toxic. Furthermore, no single approach works for all macromolecular cargo, across cell types, or in every context (e.g. cell cultures vs in vivo). This problem is exacerbated by emerging biological applications continually pushing the boundaries of required delivery efficiencies and versatility (e.g. CRISPR-Cas9 technologies). This project aims to reveal fundamental mechanisms of how to permeate cellular membranes, enabling precise control of the molecules that achieve this cell permeation, and to develop new platforms for cellular delivery. Thus, the proposed studies will significantly advance both understanding and solutions to the cell delivery problem.

项目摘要/摘要 标题：用于细胞输送应用的机理和优化内体逃生 通过精确的生物学上的细胞内递送来控制细胞 活性材料一直是探测细胞机制和治疗性的长期目标 干预措施。但是，蜂窝递送是尚未解决的问题。最多 技术仍然效率低下，对细胞具有破坏性，可能是有毒的。此外，没有一个 方法适用于所有大分子货物，跨细胞类型或在每种情况下都可以使用（例如细胞 文化与体内）。新兴的生物应用程序加剧了这个问题 不断突破所需的交付效率和多功能性的边界（例如 CRISPR-CAS9技术）。该项目旨在揭示如何的基本机制 渗透到细胞膜，以精确控制达到该细胞的分子 渗透，并开发新的平台用于蜂窝传递。因此，拟议的研究将 显着提高了细胞输送问题的理解和解决方案。