Variant-selective stereopure oligonucleotides protect against pathologies associated with C9orf72-repeat expansion in preclinical models

Variant-selective stereopure oligonucleotides protect against pathologies associated with C9orf72-repeat expansion in preclinical models. Liu Y, Dodart JC, Tran H, Berkovitch S, Braun M, Byrne M, Durbin AF, Hu XS, Iwamoto N, Jang HG, Kandasamy P, Liu F, Longo K, Ruschel J, Shelke J, Yang H, Yin Y, Donner A, Zhong Z, Vargeese C, Brown RH…

Safety, Pharmacokinetic and Pharmacodynamic Evaluation of a 2′-MOE Antisense Oligonucleotide-GalNAc3 Conjugate that Targets the Human Transmembrane Protease Serine 6 (TMPRSS6)

Safety, Pharmacokinetic and Pharmacodynamic Evaluation of a 2′-MOE Antisense Oligonucleotide-GalNAc3 Conjugate that Targets the Human Transmembrane Protease Serine 6 (TMPRSS6). Zanardi TA, Korbmacher B, Boone L, Engelhardt JA, Wang Y, Burel S, Prill B, Aghajan M, Guo S, Henry SP. J Pharmacol Exp Ther. 2021 Jan 11:JPET-AR-2020-000222 Pubmed 00