Matches in SemOpenAlex for { <https://semopenalex.org/work/W3036363721> ?p ?o ?g. }
- W3036363721 abstract "ABSTRACT RNA interference (RNAi) is an ancient biological mechanism used to defend against external invasion. It theoretically can silence any disease-related genes in a sequence-specific manner, making small interfering RNA (siRNA) a promising therapeutic modality. After a two-decade journey from its discovery, two approvals of siRNA therapeutics, ONPATTRO ® (patisiran) and GIVLAARI™ (givosiran), have been achieved by Alnylam Pharmaceuticals. Reviewing the long-term pharmaceutical history of human beings, siRNA therapy currently has set up an extraordinary milestone, as it has already changed and will continue to change the treatment and management of human diseases. It can be administered quarterly, even twice-yearly, to achieve therapeutic effects, which is not the case for small molecules and antibodies. The drug development process was extremely hard, aiming to surmount complex obstacles, such as how to efficiently and safely deliver siRNAs to desired tissues and cells and how to enhance the performance of siRNAs with respect to their activity, stability, specificity and potential off-target effects. In this review, the evolution of siRNA chemical modifications and their biomedical performance are comprehensively reviewed. All clinically explored and commercialized siRNA delivery platforms, including the GalNAc ( N -acetylgalactosamine)–siRNA conjugate, and their fundamental design principles are thoroughly discussed. The latest progress in siRNA therapeutic development is also summarized. This review provides a comprehensive view and roadmap for general readers working in the field." @default.
- W3036363721 created "2020-06-25" @default.
- W3036363721 creator A5002659900 @default.
- W3036363721 creator A5041522420 @default.
- W3036363721 creator A5062717395 @default.
- W3036363721 creator A5065773926 @default.
- W3036363721 creator A5082367379 @default.
- W3036363721 creator A5084662844 @default.
- W3036363721 creator A5089908612 @default.
- W3036363721 date "2020-06-19" @default.
- W3036363721 modified "2023-10-09" @default.
- W3036363721 title "Therapeutic siRNA: state of the art" @default.
- W3036363721 cites W1264104737 @default.
- W3036363721 cites W142058135 @default.
- W3036363721 cites W1549185695 @default.
- W3036363721 cites W1564730852 @default.
- W3036363721 cites W156663822 @default.
- W3036363721 cites W1575526898 @default.
- W3036363721 cites W1611419331 @default.
- W3036363721 cites W1675450733 @default.
- W3036363721 cites W1677186713 @default.
- W3036363721 cites W1780086376 @default.
- W3036363721 cites W1814998281 @default.
- W3036363721 cites W1844274376 @default.
- W3036363721 cites W1911340207 @default.
- W3036363721 cites W1964146045 @default.
- W3036363721 cites W1964837064 @default.
- W3036363721 cites W1966625970 @default.
- W3036363721 cites W1966973232 @default.
- W3036363721 cites W1968121484 @default.
- W3036363721 cites W1969989481 @default.
- W3036363721 cites W1972092494 @default.
- W3036363721 cites W1972637758 @default.
- W3036363721 cites W1973595732 @default.
- W3036363721 cites W1980880341 @default.
- W3036363721 cites W1982902233 @default.
- W3036363721 cites W1984131396 @default.
- W3036363721 cites W1985013843 @default.
- W3036363721 cites W1987554430 @default.
- W3036363721 cites W1988613221 @default.
- W3036363721 cites W1988809912 @default.
- W3036363721 cites W1992218065 @default.
- W3036363721 cites W1995177765 @default.
- W3036363721 cites W1995770815 @default.
- W3036363721 cites W1996397422 @default.
- W3036363721 cites W1998025926 @default.
- W3036363721 cites W2000071382 @default.
- W3036363721 cites W2001314633 @default.
- W3036363721 cites W2002516695 @default.
- W3036363721 cites W2004971567 @default.
- W3036363721 cites W2005134483 @default.
- W3036363721 cites W2008567035 @default.
- W3036363721 cites W2009816246 @default.
- W3036363721 cites W2010506716 @default.
- W3036363721 cites W2014509855 @default.
- W3036363721 cites W2014661213 @default.
- W3036363721 cites W2017003565 @default.
- W3036363721 cites W2018116983 @default.
- W3036363721 cites W2018328995 @default.
- W3036363721 cites W2019631541 @default.
- W3036363721 cites W2019818589 @default.
- W3036363721 cites W2022810557 @default.
- W3036363721 cites W2023843484 @default.
- W3036363721 cites W2023971542 @default.
- W3036363721 cites W2024102779 @default.
- W3036363721 cites W2024579314 @default.
- W3036363721 cites W2025663809 @default.
- W3036363721 cites W2027017068 @default.
- W3036363721 cites W2027319450 @default.
- W3036363721 cites W2029271422 @default.
- W3036363721 cites W2029397995 @default.
- W3036363721 cites W2029445916 @default.
- W3036363721 cites W2032288259 @default.
- W3036363721 cites W2033520317 @default.
- W3036363721 cites W2034636759 @default.
- W3036363721 cites W2048369172 @default.
- W3036363721 cites W2048809853 @default.
- W3036363721 cites W2052355985 @default.
- W3036363721 cites W2053515735 @default.
- W3036363721 cites W2055234488 @default.
- W3036363721 cites W2057080391 @default.
- W3036363721 cites W2057305507 @default.
- W3036363721 cites W2058647478 @default.
- W3036363721 cites W2060221489 @default.
- W3036363721 cites W2060295154 @default.
- W3036363721 cites W2060459188 @default.
- W3036363721 cites W2065338349 @default.
- W3036363721 cites W2065693353 @default.
- W3036363721 cites W2069904748 @default.
- W3036363721 cites W2070693578 @default.
- W3036363721 cites W2071030419 @default.
- W3036363721 cites W2071136305 @default.
- W3036363721 cites W2071206211 @default.
- W3036363721 cites W2071230707 @default.
- W3036363721 cites W2071302752 @default.
- W3036363721 cites W2071946945 @default.
- W3036363721 cites W2078890343 @default.
- W3036363721 cites W2080718344 @default.
- W3036363721 cites W2081557929 @default.
- W3036363721 cites W2083249633 @default.