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

I-Corps项目更广泛的影响/商业潜力是加速和控制基因传递，并允许研究人员找到新的解决方案来挑战疾病。基因传递，也被称为转染，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利益。利用基因疗法和免疫疗法，基因传递可以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光响应的纳米颗粒技术，具有更高的功效、可控的基因释放和更快的转染速率。这有可能大大减少转染所需的时间，以及大大减少所需的基因和/或药物的数量。因此，除了为研究人员提供通过简单的光触发器控制过程的独特能力外，这项技术还可以提供实质性的商业影响。这个I-Corps项目是一个基因传递工具，利用两种生物相容性表面活性剂，当溶解在水中时，它们会自发结合成光敏纳米颗粒。核酸（DNA/siRNA）可以装载在纳米颗粒内部，以保护核酸直到它们到达细胞内部。类似地，疏水/亲水药物分子也可以在纳米颗粒内携带。随后暴露于紫外线（UV）光下导致纳米颗粒解离，导致核酸的触发释放，然后核酸可以修饰细胞的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，我们的技术结合了更低的成本（载体成分和核酸由于效率更高）和更快的传递/转染率，可能会对整个行业产生相当大的影响。

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.