Pharmacokinetic and Pharmacodynamic Investigations of ION-353382, a Model Antisense Oligonucleotide: Using Alpha-2-Macroglobulin and Murinoglobulin Double-Knockout Mice

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Evaluation of Therapeutic Oligonucleotides for Familial Amyloid Polyneuropathy in Patient-Derived Hepatocyte-Like Cells

Evaluation of Therapeutic Oligonucleotides for Familial Amyloid Polyneuropathy in Patient-Derived Hepatocyte-Like Cells …………… Niemietz CJ, Sauer V, Stella J, Fleischhauer L, Chandhok G, Guttmann S, Avsar Y, Guo S, Ackermann EJ, Gollob J, Monia BP, Zibert A, Schmidt HH. PLoS One. 2016 Sep 1;11(9):e0161455. PubMed 00