siRNA therapeutics: a clinical reality

Since the revolutionary discovery of RNA interference (RNAi), a remarkable progress has been achieved in understanding and harnessing gene silencing mechanism; especially in small interfering RNA (siRNA) therapeutics. Despite its tremendous potential benefits, major challenges in most siRNA therapeu...

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Bibliographic Details
Published in:Science China. Life sciences 2020-04, Vol.63 (4), p.485-500
Main Authors: Saw, Phei Er, Song, Er-Wei
Format: Article
Language:English
Subjects:
Amyloidosis
Biomedical and Life Sciences
Clinical trials
Disease
Drug dosages
FDA approval
Federal regulation
Gene expression
Gene silencing
Hepatitis
Life Sciences
Liver
Proteins
Review
RNA-mediated interference
siRNA
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Summary:Since the revolutionary discovery of RNA interference (RNAi), a remarkable progress has been achieved in understanding and harnessing gene silencing mechanism; especially in small interfering RNA (siRNA) therapeutics. Despite its tremendous potential benefits, major challenges in most siRNA therapeutics remains unchanged—safe, efficient and target oriented delivery of siRNA. Twenty years after the discovery of RNAi, siRNA therapeutics finally charts its way into clinics. As we journey through the decades, we reminisce the history of siRNA discovery and its application in a myriad of disease treatments. Herein, we highlight the breakthroughs in siRNA therapeutics, with special feature on the first FDA approved RNAi therapeutics Onpattro (Patisiran) and the consideration of effective siRNA delivery system focusing on current siRNA nanocarrier in clinical trials. Lastly, we present some challenges and multiple barriers that are yet to be fully overcome in siRNA therapeutics.
ISSN:1674-7305
1869-1889
DOI:10.1007/s11427-018-9438-y