First-in-class fully-biodegradable DNA delivery system for biomedical research and safe gene therapy

外源DNA分子在细胞内的转移是生物医学研究中经常使用的一项重要的实验室技术，也是一种有希望的治疗方法，用于治疗由基因故障引起的疾病，如囊性纤维化、遗传性眼病和几种癌症。除了有争议的基于病毒的载体外，DNA递送可以使用具有复杂DNA分子并将其转移到细胞质中的能力的化学试剂来实现。然而，体内递送在很大程度上受到试剂毒性、DNA大小和靶细胞/组织的限制。在这项可行性研究中，DELIVERICS将研究一种新的可生物降解递送系统，以实现体内细胞内DNA的安全转移。这种独特的工具将解决研发和制药行业存在的商业机会，其中研发研究和基于dna的生物制药的治疗递送需要先进的输送系统。

The transfer of foreign DNA molecules inside cells is an essential laboratory technique routinely used in biomedical research and a promissing therapeutic approach to treat disorders caused by malfunctioning genes such as cystic fibrosis, inherited eye diseases and several kinds of cancer. Apart from the controversial virus-based carriers, DNA delivery can be achieved using chemical reagents with the ability to complex DNA molecules and translocate them into the cell cytoplasm. However, in vivo delivery is largely limited by the reagent toxicity, the DNA size and the target cell / tissue. In this feasibility study, DELIVERICS will investigate a novel biodegradable delivery system to achieve safe transfer of DNA inside cells in vivo. This unique tool will address a commercial opportunity that exists both in the R&D and Pharma sectors, where advanced delivery systems are required for R&D studies and for the therapeutic delivery of DNA-based biopharmaceuticals.