Matches in SemOpenAlex for { <https://semopenalex.org/work/W2912498563> ?p ?o ?g. }
- W2912498563 endingPage "265" @default.
- W2912498563 startingPage "233" @default.
- W2912498563 abstract "Although chemical modifications of the nucleic acid backbone, sugar, and base have imparted drug-like properties to small interfering RNA (siRNA), it was not until recently that systemic delivery at therapeutically acceptable doses was enabled. Use of ionizable lipid nanoparticles (iLNPs) and covalent conjugation to N-acetylgalactosamine (GalNAc) resulted in effective delivery of siRNAs to liver. Here, we discuss the development of these delivery technologies and describe how chemical modifications result in siRNAs that do not trigger the innate immune response and that are systemically stable. We highlight the findings from about 25 siRNA-based drugs targeting diseases originating from liver in various stages of clinical testing with a focus on several from Alnylam's clinical pipeline. In this pipeline, the clinically validated iLNP formulation by intravenous administration and GalNAc-conjugated delivery by subcutaneous administration make possible infrequent dosing of therapeutic siRNAs to several hitherto “undruggable” molecular targets, realizing the promise of RNA interference (RNAi)-based drugs to benefit patients." @default.
- W2912498563 created "2019-02-21" @default.
- W2912498563 creator A5085203302 @default.
- W2912498563 creator A5091111026 @default.
- W2912498563 date "2019-02-11" @default.
- W2912498563 modified "2023-09-28" @default.
- W2912498563 title "Liver-targeted RNAi Therapeutics: Principles and Applications" @default.
- W2912498563 cites W1545841075 @default.
- W2912498563 cites W1549185695 @default.
- W2912498563 cites W1611419331 @default.
- W2912498563 cites W1647075334 @default.
- W2912498563 cites W1808439685 @default.
- W2912498563 cites W1967317528 @default.
- W2912498563 cites W1985638596 @default.
- W2912498563 cites W1996347799 @default.
- W2912498563 cites W1996830006 @default.
- W2912498563 cites W2001314633 @default.
- W2912498563 cites W2008161734 @default.
- W2912498563 cites W2009816246 @default.
- W2912498563 cites W2018328995 @default.
- W2912498563 cites W2021905258 @default.
- W2912498563 cites W2027319450 @default.
- W2912498563 cites W2031883984 @default.
- W2912498563 cites W2034139519 @default.
- W2912498563 cites W2057519110 @default.
- W2912498563 cites W2060221489 @default.
- W2912498563 cites W2060256979 @default.
- W2912498563 cites W2060433961 @default.
- W2912498563 cites W2065338349 @default.
- W2912498563 cites W2065603844 @default.
- W2912498563 cites W2068056297 @default.
- W2912498563 cites W2078775246 @default.
- W2912498563 cites W2078890343 @default.
- W2912498563 cites W2079264126 @default.
- W2912498563 cites W2080718344 @default.
- W2912498563 cites W2086919915 @default.
- W2912498563 cites W2092222037 @default.
- W2912498563 cites W2093206703 @default.
- W2912498563 cites W2095151181 @default.
- W2912498563 cites W2096917691 @default.
- W2912498563 cites W2100451532 @default.
- W2912498563 cites W2100615745 @default.
- W2912498563 cites W2105774848 @default.
- W2912498563 cites W2106885643 @default.
- W2912498563 cites W2116424844 @default.
- W2912498563 cites W2121583027 @default.
- W2912498563 cites W2123555579 @default.
- W2912498563 cites W2133313719 @default.
- W2912498563 cites W2143743183 @default.
- W2912498563 cites W2149871164 @default.
- W2912498563 cites W2149896139 @default.
- W2912498563 cites W2156955518 @default.
- W2912498563 cites W2165586566 @default.
- W2912498563 cites W2170468945 @default.
- W2912498563 cites W2292189335 @default.
- W2912498563 cites W2314304229 @default.
- W2912498563 cites W2322913813 @default.
- W2912498563 cites W2336471513 @default.
- W2912498563 cites W2409156875 @default.
- W2912498563 cites W2409777939 @default.
- W2912498563 cites W2496344419 @default.
- W2912498563 cites W2515690407 @default.
- W2912498563 cites W2531015819 @default.
- W2912498563 cites W2555483953 @default.
- W2912498563 cites W2557045419 @default.
- W2912498563 cites W2559991352 @default.
- W2912498563 cites W2568306867 @default.
- W2912498563 cites W2595169180 @default.
- W2912498563 cites W2600400434 @default.
- W2912498563 cites W2604979510 @default.
- W2912498563 cites W2621040239 @default.
- W2912498563 cites W2624827575 @default.
- W2912498563 cites W2630192492 @default.
- W2912498563 cites W2734498030 @default.
- W2912498563 cites W2754443480 @default.
- W2912498563 cites W2755631183 @default.
- W2912498563 cites W2756425540 @default.
- W2912498563 cites W2757504452 @default.
- W2912498563 cites W2757654367 @default.
- W2912498563 cites W2768219381 @default.
- W2912498563 cites W2770098637 @default.
- W2912498563 cites W2775056759 @default.
- W2912498563 cites W2781906140 @default.
- W2912498563 cites W2785178319 @default.
- W2912498563 cites W2789738592 @default.
- W2912498563 cites W2793722319 @default.
- W2912498563 cites W2794164641 @default.
- W2912498563 cites W2795531081 @default.
- W2912498563 cites W2795823418 @default.
- W2912498563 cites W2800649167 @default.
- W2912498563 cites W2801179771 @default.
- W2912498563 cites W2801612759 @default.
- W2912498563 cites W2802881274 @default.
- W2912498563 cites W2803566305 @default.
- W2912498563 cites W2810909862 @default.
- W2912498563 cites W4251060592 @default.
- W2912498563 cites W804074280 @default.
- W2912498563 doi "https://doi.org/10.1039/9781788015714-00233" @default.