Novel method of in vivo gene delivery using a needle-free system

基因操作技术的最新进展为遗传病的治疗开辟了新的机会。尽管在开发可用于活体基因编辑的潜在工具方面取得了显著进展，但将这些工具(如外源DNA分子)输送到细胞内的方法仍然不发达。在目前的提案中，我们将使用一家加拿大公司发明的无针头递送系统，该系统利用空气压力将药物输送到组织深处。我们将开发方法，在细胞内传递DNA-化学混合物的组合。高压与DNA化学复合体的结合在以前还没有尝试过。如果成功，这种新的方法将扩大现有系统的使用，使其成为现代生物医学研究的重要工具。这不仅将使研究界受益，还将创造范围，扩大公司的业务，并最终为加拿大经济做出贡献。

Recent advances in gene manipulation techniques have opened up new opportunities for the treatment ofhereditary diseases. Although remarkable progresses have been made in the development of the potential toolswhich can be used for gene editing in the living organisms, the methods to deliver these tools (e.g. foreignDNA molecules) inside the cells are still under-developed. In the current proposal we will use a needle-freedelivery system invented by a Canadian company that uses air pressure to deliver medicine deep inside thetissue. We will develop methodologies to deliver combination of DNA-chemical mixtures inside the cells. Thecombination of high air pressure together with DNA-chemical complexes has not been tried before. Ifsuccessful, this novel approach will expand the use of the existing system making it an essential tool in modernbiomedical research. This will not only benefit the research community, but also create scopes to expand thebusiness of the company and eventual contribution to Canadian Economy.