A novel framework for nanomedicine development

纳米医学开发的新框架

• 批准号: 10715013
• 负责人: Stefan Wilhelm
• 金额: $ 37.33万
• 依托单位: UNIVERSITY OF OKLAHOMA
• 依托单位国家: 美国
• 财政年份: 2023
• 资助国家: 美国
• 起止时间: 2023-09-01 至 2028-07-31
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10715013
• 关键词: Area; Cells; Development; Diagnostic; Effectiveness; Goals; Kinetics; Medicine; Methods; Mission; Muscle Fibers; Myopathy; Nanotechnology; National Institute of General Medical Sciences; Nucleic Acids; Organ; Pharmaceutical Preparations; Research; Safety; Skeletal Muscle; Therapeutic; Therapeutic Uses; Tissues; bioimaging; biological systems; in vivo; nanomedicine; nanoparticle; nanoparticle delivery; nanoscale; novel; programs; small molecule; spatiotemporal; ultra high resolution

Project Summary Nanomedicine is a research field centered around applying materials in the nanoscale size range (i.e. nanoparticles) as therapeutics and diagnostics. However, a major challenge is the safe, efficient, and effective delivery of nanoparticles to biological systems, such as cells, tissues, and organs. In this project, we will introduce and explore a novel framework for nanomedicine development with the ultimate goal of efficiently delivering non- viral nanoparticles to cells and tissues for safe and effective applications. Our proposed research program has three main strands: (i) we will develop novel nanoparticles for the safe, efficient, and effective delivery of small molecules and nucleic acid payloads to skeletal muscle cells; (ii) we will develop novel methods for super- resolution bioimaging to study the mechanisms and kinetics of nanoparticle spatiotemporal transport in biological systems; (iii) we will quantify safety, efficiency, and effectiveness of nanoparticle in vivo delivery. Using this novel framework, our long-term scientific goal is to create a major breakthrough in nanomedicine by developing safer, more efficient, and more effective nanoparticles for the non-viral delivery of small molecule-based drugs and nucleic acid payloads to enable therapeutic applications in skeletal muscle disorders.

项目摘要 纳米医学是一个研究领域，其中心是应用纳米尺寸范围内的材料（即， 纳米颗粒）作为治疗剂和诊断剂。然而，一个主要的挑战是安全，高效和有效的 将纳米颗粒递送至生物系统，例如细胞、组织和器官。在这个项目中，我们将介绍 并探索纳米医学发展的新框架，其最终目标是有效地提供非药物， 将病毒纳米颗粒应用于细胞和组织，以实现安全有效的应用。我们提出的研究计划 三个主要方面：（i）我们将开发新型纳米颗粒，用于安全，高效和有效地输送小分子药物。 分子和核酸有效载荷骨骼肌细胞;（ii）我们将开发新的方法， 分辨率生物成像，研究纳米粒子在生物体内时空运输的机制和动力学 系统;（iii）我们将量化纳米颗粒体内递送的安全性、效率和有效性。使用该新颖 框架，我们的长期科学目标是通过开发更安全， 更高效、更有效的纳米颗粒，用于小分子药物的非病毒递送， 核酸有效载荷，从而能够在骨骼肌病症中进行治疗应用。