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Uniqueness, Advantages, Challenges, Solutions, and Perspectives in Therapeutics Applying RNA Nanotechnology

期刊

NUCLEIC ACID THERAPEUTICS
卷 22, 期 4, 页码 226-245

出版社

MARY ANN LIEBERT, INC
DOI: 10.1089/nat.2012.0350

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资金

  1. NIH [R01 EB003730, R01 EB012135, U01 CA151648, R01 GM059944]
  2. NIH Nanomedicine Development Center [Phi29]
  3. DNA Packaging Motor for Nanomedicine, through the NIH Roadmap for Medical Research [PN2 EY 018230]

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The field of RNA nanotechnology is rapidly emerging. RNA can be manipulated with the simplicity characteristic of DNA to produce nanoparticles with a diversity of quaternary structures by self-assembly. Additionally RNA is tremendously versatile in its function and some RNA molecules display catalytic activities much like proteins. Thus, RNA has the advantage of both worlds. However, the instability of RNA has made many scientists flinch away from RNA nanotechnology. Other concerns that have deterred the progress of RNA therapeutics include the induction of interferons, stimulation of cytokines, and activation of other immune systems, as well as short pharmacokinetic profiles in vivo. This review will provide some solutions and perspectives on the chemical and thermodynamic stability, in vivo half-life and biodistribution, yield and production cost, in vivo toxicity and side effect, specific delivery and targeting, as well as endosomal trapping and escape.

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