Investigating the pharmacodynamic durability of GalNAc-siRNA conjugates.
Brown CR, Gupta S, Qin J, Racie T, He G, Lentini S, Malone R, Yu M, Matsuda S, Shulga-Morskaya S, Nair AV, Theile CS, Schmidt K, Shahraz A, Goel V, Parmar RG, Zlatev I, Schlegel MK, Nair JK, Jayaraman M, Manoharan M, Brown D, Maier MA, Jadhav V.
Brown CR, et al.
Nucleic Acids Res. 2020 Dec 2;48(21):11827-11844. doi: 10.1093/nar/gkaa670.
Nucleic Acids Res. 2020.
PMID: 32808038
Free PMC article.
One hallmark of trivalent N-acetylgalactosamine (GalNAc)-conjugated siRNAs is the remarkable durability of silencing that can persist for months in preclinical species and humans. ...Identical siRNAs delivered in lipid nanoparticles or as GalNAc conjugates were dose …
One hallmark of trivalent N-acetylgalactosamine (GalNAc)-conjugated siRNAs is the remarkable durability of silencing that can persist …