Engineering biodegradable polymers for gene delivery

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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.