Towards next generation antisense oligonucleotides: mesylphosphoramidate modification improves therapeutic index and duration of effect of gapmer antisense oligonucleotides.

Autor: Anderson BA; Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA., Freestone GC; Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA., Low A; Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA., De-Hoyos CL; Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA., Iii WJD; Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA., Østergaard ME; Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA., Migawa MT; Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA., Fazio M; Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA., Wan WB; Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA., Berdeja A; Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA., Scandalis E; Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA., Burel SA; Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA., Vickers TA; Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA., Crooke ST; Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA., Swayze EE; Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA., Liang X; Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA., Seth PP; Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA.

Publikováno v: Nucleic acids research [Nucleic Acids Res] 2021 Sep 20; Vol. 49 (16), pp. 9026-9041.