Development of an Integrated Microfluidic Platform for the Identification of Therapeutic Peptides for Unmet Medical Needs

制药行业已经认识到细胞渗透肽在治疗目前无法治愈的疾病方面的重要性。事实上，多肽可以与其他类型的分子不能相互作用的靶点相互作用，例如在许多疾病的发生和发展中发挥关键作用的蛋白质-蛋白质相互作用。然而，多肽也有很大的局限性，阻碍了它们作为药物的使用。其中之一是它们无法有效地与细胞内靶点相互作用，留下了许多未得到满足的医疗需求。我们建议开发一个综合平台来生成和筛选多肽文库，以快速和经济高效地为那些未开发的细胞内靶点识别新药。在拟议的研究的第一阶段，我们将专注于建立这样一个平台，在第二阶段，我们将使用与癌症和囊性纤维化相关的两个不同的细胞内靶点来验证它。该项目的成功结果将导致药物发现领域的重大进步，因为目前的多肽技术只能访问细胞外靶点，而忽略了75%位于细胞内的靶点。因此，我们的技术有可能为患者提供改变生命的药物，用于目前无法治疗的疾病或治疗选择有限的疾病。

Pharmaceutical industries have recognised the importance of cell-permeable peptides to cure currently untreatable diseases. In fact, peptides can interact with targets that other type of molecules cannot, such as protein-protein interactions which play crucial roles in the generation and progression of many diseases. However, peptides also have major limitations that hamper their use as drugs. Among these is their inability to efficiently interact with intracellular targets leaving many unmet medical needs. We propose to develop an integrated platform to generate and screen libraries of peptides to rapidly and cost-effectively identify new drugs for those untapped intracellular targets. In the first phase of the proposed research, we will focus our efforts on building such a platform, and in the second phase we will validate it using two different intracellular targets with relevance in cancer and cystic fibrosis. A successful outcome to this project will result in a significant advance in the drug-discovery field as current peptide technologies can only access extracellular targets, ignoring those 75% located intracellularly. Therefore, our technology has the potential to provide patients with life-changing medicines for currently untreatable diseases or for diseases with limited therapeutic options available.