Clinical advances of siRNA therapeutics

Small interfering RNA (siRNA) enables efficient target gene silencing by employing a RNA interference (RNAi) mechanism, which can compromise gene expression and regulate gene activity by cleaving mRNA or repressing its translation. Twenty years after the discovery of RNAi in 1998, ONPATTRO™ (patisir...

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Veröffentlicht in:The journal of gene medicine Jg. 21; H. 7; S. e3097 - n/a
Hauptverfasser: Hu, Bo, Weng, Yuhua, Xia, Xin‐Hua, Liang, Xing‐jie, Huang, Yuanyu
Format: Journal Article
Sprache:Englisch
Veröffentlicht: England Wiley Periodicals Inc 01.07.2019
Schlagworte:
Gene expression
Gene silencing
Gene therapy
RNA interference
RNA-mediated interference
siRNA
siRNA clinical study
siRNA delivery
gene silencing
siRNA delivery
siRNA clinical study
siRNA
RNA interference
ISSN:1099-498X, 1521-2254, 1521-2254
Online-Zugang:Volltext
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Zusammenfassung:Small interfering RNA (siRNA) enables efficient target gene silencing by employing a RNA interference (RNAi) mechanism, which can compromise gene expression and regulate gene activity by cleaving mRNA or repressing its translation. Twenty years after the discovery of RNAi in 1998, ONPATTRO™ (patisiran) (Alnylam Pharmaceuticals, Inc.), a lipid formulated siRNA modality, was approved for the first time by United States Food and Drug Administration and the European Commission in 2018. With this milestone achievement, siRNA therapeutics will soar in the coming years. Here, we review the discovery and the mechanisms of RNAi, briefly describe the delivery technologies of siRNA, and summarize recent clinical advances of siRNA therapeutics.
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ISSN:1099-498X
1521-2254
1521-2254
DOI:10.1002/jgm.3097