Engineering biodegradable polymers for gene delivery

基因治疗是治疗几种重要疾病的有效方法，因为它可以解决问题的根本原因，而不是缓解当前药物的典型疾病症状。对于成功的基因治疗程序，有必要使用特定的传递系统，以便在DNA分子中发现的基因可以由患者自身的细胞表达。使用病毒将基因传递到细胞是流行的，但是当病毒用于患者细胞时会引起各种问题。使用合成材料而不是病毒更可取，因为它们对细胞造成的问题相对较少。然而，在基因传递方面，它们不如病毒有效。该项目将开发新的合成运载系统，以改善现有系统的性能。我们将设计新材料，使它们能够有效地将DNA分子呈递给患者的细胞，并允许更有效地使用所递送的DNA治疗剂。该项目的成功完成将导致在临床环境中更好地使用基因疗法治疗各种疾病。

Gene therapy is a powerful approach to treatment of several important diseases since it can address the root cause of the problem rather than alleviate the disease symptoms that are typical with current drugs. For a successful gene therapy procedure, it is necessary to use specific delivery systems so that the genes found in DNA molecules can be expressed by patients' own cells. Using viruses to deliver the genes to the cells is popular, but viruses cause a variety of problems when they are used on patient cells. Using synthetic materials, rather than viruses, is more desirable since they have cause relatively less problems on the cells. However, they are not as effective as viruses when it comes to gene delivery. This project will develop new synthetic delivery systems that will improve the performance of current systems. We will engineer new materials so that they can effectively present the DNA molecules to patients' cells and allow more effective use of the delivered DNA therapeutics. Successful completion of the project will lead to better use of gene therapy in a clinical setting for a wide range of diseases.