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Intracellular Transport and Unpacking of Polyplex Nanoparticlesстатья Глава в книге
- Авторы: Rosenkranz AA, Khramtsov YV, Ulasov AV, Rodichenko N., Sobolev AS
- Сборник: Handbook of Clinical Nanomedicine – From Bench to Bedside
- Том: 2
- Глава в коллективной монографии
- Год издания: 2016
- Место издания: CRC PRESS-TAYLOR & FRANCIS GROUP Singapore
- Первая страница: 828
- Последняя страница: 847
- DOI: 10.1201/b19910
- Аннотация: Gene therapy has recently been transformed from a basic research to a commercially available method of treatment. Use of viral gene delivery methods has generally been better in terms of efficiency, but they have safety concerns. Non-viral vectors, on the other hand, offer safety but have often been disappointing in terms of efficacy of nuclear delivery. Polyplexes are typical examples of nanoparticles designed for transport of nucleic acids into cells, and some of them have been studied in clinical trials. The approach described here can be useful for rational design of polyplexes by addressing the individual rate-limiting steps such as rate of polyplex entry and their unpacking into the acidifying endocytotic compartments. Moreover, the approach provides quantitative characteristics of transport and state of polyplex nanoparticles within living cells and helps to interpret the data. Using this information, one will be able to choose optimal DNA-delivering constructs for a particular cell type and to optimize them by modifying their cell specificity as well as features of their intracellular transport. In the future it will provide flexible methods of gene delivery targeted for the particular patient.
- Добавил в систему: Розенкранц Андрей Александрович