Restricted accessResearch articleFirst published online 2022-02
17th Annual Meeting of the Oligonucleotide Therapeutics Society: A Tribute to Bob Letsinger,Progress in N = 1 Treatments,and Successful First In-Humans CRISPR Trials
FireA, XuS, MontgomeryMK, KostasSA, DriverSE and MelloCC. (1998). Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature, 391:806–811.
2.
ElbashirSM, HarborthJ, LendeckelW, YalcinA, WeberK and TuschlT. (2001). Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature, 411:494–498.
3.
KleinD, GoldbergS, TheileCS, DambraR, HaskellK, KuharE, LinT, ParmarR, ManoharanM, et al. (2021). Centyrin ligands for extrahepatic delivery of siRNA. Mol Ther, 29:2053–2066.
4.
BrownCR, GuptaS, QinJ, RacieT, HeG, LentiniS, MaloneR, YuM, MatsudaS, et al. (2020). Investigating the pharmacodynamic durability of GalNAc-siRNA conjugates. Nucleic Acids Res, 48:11827–11844.
5.
JacksonLA, AndersonEJ, RouphaelNG, RobertsPC, Makhene M, ColerRN, McCulloughMP, ChappellJD, DenisonMR, et al. (2020). An mRNA vaccine against SARS-CoV-2—preliminary report. N Engl J Med, 383:1920–1931.
6.
MulliganMJ, LykeKE, KitchinN, AbsalonJ, GurtmanA, LockhartS, NeuzilK, RaabeV, BaileyR, et al. (2020). Phase I/II study of COVID-19 RNA vaccine BNT162b1 in adults. Nature, 586:589–593.
7.
GillmoreJD, GaneE, TaubelJ, KaoJ, FontanaM, MaitlandML, SeitzerJ, O'ConnellD, WalshKR, et al. (2021). CRISPR-Cas9 in vivo gene editing for transthyretin amyloidosis. N Engl J Med, 385:493–502.
