期刊
JOURNAL OF CONTROLLED RELEASE
卷 218, 期 -, 页码 94-113出版社
ELSEVIER SCIENCE BV
DOI: 10.1016/j.jconrel.2015.09.066
关键词
Local delivery; Gene silencing; siRNA
资金
- NIBIB NIH HHS [NIH R01 EB019409, R21 EB012750, R01 EB019409] Funding Source: Medline
The discovery of RNAi in the late 1990s unlocked a new realm of therapeutic possibilities by enabling potent and specific silencing of theoretically any desired genetic target. Better elucidation of the mechanism of action, the impact of chemical modifications that stabilize and reduce nonspecific effects of siRNA molecules, and the key design considerations for effective delivery systems has spurred progress toward developing clinically-successful siRNA therapies. A logical aim for initial siRNA translation is local therapies, as delivering siRNA directly to its site of action helps to ensure that a sufficient dose reaches the target tissue, lessens the potential for off-target side effects, and circumvents the substantial systemic delivery barriers. While locally injected or topically applied siRNA has progressed into numerous clinical trials, an enormous opportunity exists to develop sustained-release, local delivery systems that enable both spatial and temporal control of gene silencing. This review focuses on material platforms that establish both localized and controlled gene silencing, with emphasis on the systems that show most promise for clinical translation. (C) 2015 Elsevier B.V. All rights reserved.
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