I-Corps: Photo-controlled Nanoparticles for Gene and Drug Delivery

I-Corps：用于基因和药物输送的光控纳米颗粒

• 批准号: 1758225
• 负责人: C. Ted Lee, Jr.
• 金额: $ 5万
• 依托单位: University of Southern California
• 依托单位国家: 美国
• 项目类别: Standard Grant
• 财政年份: 2018
• 资助国家: 美国
• 起止时间: 2018-01-01 至 2019-07-31
• 项目状态: 已结题
• 来源: https://www.nsf.gov/awardsearch/showAward?AWD_ID=1758225&HistoricalAwards=false
• 关键词: Corps; Photo; controlled; Nanoparticles; Gene

The broader impact/commercial potential of this I-Corps project is to accelerate and control gene delivery, and to allow researchers to find novel solutions to challenging diseases. Gene delivery, also known as transfection, is a promising approach to treat diseases such as cancer and, thus, could provide a profound benefit to society. Using gene therapy and immune-therapy, gene delivery allows for the efficient introduction of a new gene into cells. The specific technology employed offers a photo-responsive, nanoparticle-based technique with the advantages of higher efficacy, controlled gene-release, and faster transfection rates. This has the potential to dramatically decrease the time required for transfection, as well as greatly reducing the required amount of gene and/or drug. Thus, along with providing researchers the unique ability to control the process through a simple light trigger, this technology could provide substantial commercial impact.This I-Corps project is a gene delivery tool that utilizes two biocompatible surfactants which associate spontaneously into light-sensitive nanoparticles when dissolved in water. Nucleic acids (DNA/siRNA) can be loaded inside of the nanoparticles to protect the nucleic acids until they reach the interior of cells. Similarly, hydrophobic/hydrophilic drug molecules can also be carried within the nanoparticles. Subsequent exposure to ultraviolet (UV) light leads to nanoparticle dissociation, resulting in the triggered release of nucleic acids, which can then modify the DNA of the cell or stop disease-associated protein production. Researchers would be able to utilize this gene delivery technology in two different ways: (1) substitution of new genes to the cell, or (2) inactivation of existing genes. The combination of lower costs (of both the carrier components and the nucleic acids due to higher efficiencies) and faster delivery/transfection rates with our technology compared to current gene-delivery technologies could have a considerable impact throughout the industry.

这个i-Corps项目的更广泛的影响/商业潜力是加速和控制基因传递，并使研究人员能够找到具有挑战性的疾病的新解决方案。基因传递，也被称为转基因，是治疗癌症等疾病的一种很有前途的方法，因此可以为社会带来深远的好处。使用基因治疗和免疫治疗，基因传递允许有效地将新基因引入细胞。所采用的特定技术提供了一种基于纳米颗粒的光响应技术，具有更高的效率、可控的基因释放和更快的转染率。这有可能极大地减少转基因所需的时间，以及极大地减少所需的基因和/或药物的量。因此，除了为研究人员提供通过简单的光触发控制这一过程的独特能力外，这项技术还可以带来实质性的商业影响。这项i-Corps项目是一种基因传递工具，它利用两种生物兼容的表面活性剂，它们在溶于水时自发结合成光敏纳米颗粒。核酸(DNA/siRNA)可以被装载在纳米颗粒内，以保护核酸，直到它们到达细胞内部。同样，疏水/亲水药物分子也可以携带在纳米颗粒中。随后暴露在紫外线(UV)下会导致纳米颗粒解离，从而触发核酸的释放，然后核酸可以修改细胞的DNA或停止与疾病相关的蛋白质的产生。研究人员将能够以两种不同的方式利用这种基因传递技术：(1)将新基因替换到细胞中，或(2)使现有基因失活。与目前的基因传递技术相比，我们的技术成本更低(载体组件和核酸的成本都更高)和更快的传递/转染率相结合，可能会对整个行业产生相当大的影响。