A novel framework for nanomedicine development

项目摘要 纳米医学是以应用纳米尺度范围内的材料为中心的研究领域。 纳米粒子)作为治疗学和诊断学。然而，一个主要的挑战是安全、高效和有效的 将纳米颗粒输送到生物系统，如细胞、组织和器官。在这个项目中，我们将介绍 并探索一种新的纳米医学开发框架，最终目标是高效地提供 病毒纳米粒子到细胞和组织的安全和有效的应用。我们提出的研究计划已经 三个主要方面：(I)我们将开发新的纳米颗粒，以安全、高效和有效地运送小分子药物 分子和核酸负载到骨骼肌细胞；(Ii)我们将开发新的方法来超 分辨率生物成像研究纳米粒子在生物体内的时空传输机制和动力学 (3)我们将量化体内纳米颗粒的安全性、效率和有效性。用这本小说 我们的长期科学目标是通过开发更安全、更安全的 更高效、更有效的纳米颗粒，用于小分子药物的非病毒传递和 核酸有效载荷使骨骼肌疾病的治疗应用成为可能。

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.