期刊
BIOTECHNOLOGY ADVANCES
卷 32, 期 4, 页码 831-843出版社
PERGAMON-ELSEVIER SCIENCE LTD
DOI: 10.1016/j.biotechadv.2013.08.020
关键词
Gene therapy; RNA interference (RNAi); Small-interfering RNA (siRNA); Gene delivery; Nanoparticles
资金
- Major State Basic Research Development Program of China (973 Program) [2013CB733802, 2014CB744503]
- National Science Foundation of China (NSFC) [81101101, 81371596, 51273165, 51203177]
- Key Project of Chinese Ministry of Education [212149]
- Fundamental Research Funds for the Central Universities [2013121039]
- Intramural Research Program (IRP) of the National Institute of Biomedical Imaging and Bioengineering (NIBIB), National Institutes of Health (NIH)
Gene therapy is a promising strategy to treat various genetic and acquired diseases. Small interfering RNA (siRNA) is a revolutionary tool for gene therapy and the analysis of gene function. However, the development of a safe, efficient, and targetable non-viral siRNA delivery system remains a major challenge in gene therapy. An ideal delivery system should be able to encapsulate and protect the siRNA cargo from serum proteins, exhibit target tissue and cell specificity, penetrate the cell membrane, and release its cargo in the desired intracellular compartment. Nanomedicine has the potential to deal with these challenges faced by siRNA delivery. The unique characteristics of rigid nanoparticles mostly inorganic nanoparticles and allotropes of carbon nanomaterials, including high surface area, facile surface modification, controllable size, and excellent magnetic/optical/electrical properties, make them promising candidates for targeted siRNA delivery. In this review, recent progresses on rigid nanoparticle-based siRNA delivery systems will be summarized. Published by Elsevier Inc.
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