Nanofabricated Particles for Biolistic Delivery

描述（由申请人提供）：本研究项目的目标是继续开发一系列新型纳米制造颗粒，作为载体，通过粒子加速方法输送各种不同类型的生物活性分子（DNA， RNA，蛋白质，肽）和纳米颗粒传感器。这些粒子是由纳米颗粒、聚合物和生物分子通过一层一层的技术控制组装而成的，其中核心层和后续层分别为最终粒子添加特定的功能。这些纳米复合粒子将提高目前使用的生物过程的效率和可靠性，并使新的生物传递应用成为可能。这些包括将RNAi、反义寡核苷酸、多肽和传感器输送到活细胞和组织中，用于各种生命科学研究应用和人类治疗干预。要实现的关键技术目标包括纳米制造颗粒生产的制造和工艺开发步骤的发展，这些颗粒在各种模型系统应用中的增强功效的文件演示，以及具有特定功能的化学不同颗粒家族的合成和表征。将核酸和多肽输送到感兴趣的细胞和组织的能力是基本的和必要的技术成就之一，在某种程度上是分子生物学、基因治疗和相关领域的基础。粒子加速方法，也被称为生物递送，是几种常规使用的递送方法之一。对这些输送方法中的任何一种的改进都与人类健康有关，因为核酸（如DNA）的输送往往是医学疾病研究和发展的一个重要步骤。我们将开发的纳米制造载体粒子将显著提高生物传递过程的功效，并使其在目前尚未满足需求的新应用中得到应用。

DESCRIPTION (provided by applicant): The goal of this research project is the continued development of a family of novel nanofabricated particles for use as carriers to deliver a variety of different types of bioactive molecules (DNA, RNA, proteins, peptides) and nanoparticle sensors via particle acceleration methods. The particles are fabricated using the controlled assembly of nanoparticles, polymers and biomolecules via a layer-by-layer technique where the core and subsequent layer each add specific functionality to the final particle. These nanocomposite particles will increase the efficiency and reliability of the currently used biolistic process, and enable novel biolistic delivery applications. These include the delivery of RNAi, antisense oligonucleotides, peptides, and sensors to living cells and tissues for a variety of life science research applications, and for human therapeutic interventions. The key technical objectives to be achieved include the development of manufacturing and process development steps for the production of nanofabricated particles, the documented demonstration of the enhanced efficacy of these particles in a variety of model system applications, and synthesis and characterization of a family of chemically distinct particles with specific functionality. The ability to deliver nucleic acids and polypeptides to cells and tissues of interest is one of the fundamental and required technical achievements that has, in part, been at the foundation of molecular biology, gene therapy and related fields. Particle acceleration methods, also referred to as biolistic delivery, is one of several delivery methods in routine use. Improvements to any of these delivery methods is of relevance to human health because the delivery of nucleic acids, such as DNA, is frequently an essential step in the research and development of medical diseases. The nanofabricated carrier particles we will develop will significantly improve the efficacy of the biolistic delivery process and enable its use in novel applications where currently there is an unmet need.