Rekindling RNAi Therapy: Materials Design Requirements for In Vivo siRNA Delivery

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With the recent FDA approval of the first siRNA-derived therapeutic, RNA interference (RNAi)-mediated gene therapy is undergoing a transition from research to the clinical space. The primary obstacle to realization of RNAi therapy has been the delivery of oligonucleotide payloads. Therefore, the main aims is to identify and describe key design features needed for nanoscale vehicles to achieve effective delivery of siRNA-mediated gene silencing agents in vivo. The problem is broken into three elements: 1) protection of siRNA from degradation and clearance; 2) selective homing to target cell types; and 3) cytoplasmic release of the siRNA payload by escaping or bypassing endocytic uptake. The in vitro and in vivo gene silencing efficiency values that have been reported in publications over the past decade are quantitatively summarized by material type (lipid, polymer, metal, mesoporous silica, and porous silicon), and the overall trends in research publication and in clinical translation are discussed to reflect on the direction of the RNAi therapeutics field.
Publisher
WILEY-V C H VERLAG GMBH
Issue Date
2019-12
Language
English
Article Type
Review
Citation

ADVANCED MATERIALS, v.31, no.49, pp.1903637

ISSN
0935-9648
DOI
10.1002/adma.201903637
URI
http://hdl.handle.net/10203/270016
Appears in Collection
BiS-Journal Papers(저널논문)
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