Cationic Amphiphilic Drugs Boost the Lysosomal Escape of Small Nucleic Acid Therapeutics in a Nanocarrier-Dependent Manner

Cationic Amphiphilic Drugs Boost the Lysosomal Escape of Small Nucleic Acid Therapeutics in a Nanocarrier-Dependent Manner …………… Van de Vyver T, Bogaert B, De Backer L, Joris F, Guagliardo R, Van Hoeck J, Merckx P, Van Calenbergh S, Ramishetti S, Peer D, Remaut K, De Smedt SC, Raemdonck K. ACS Nano. 2020 Apr 28;14(4):4774-4791. PubMed

Population Pharmacokinetic-Pharmacodynamic Modeling of Inotersen, an Antisense Oligonucleotide for Treatment of Patients with Hereditary Transthyretin Amyloidosis

Population Pharmacokinetic-Pharmacodynamic Modeling of Inotersen, an Antisense Oligonucleotide for Treatment of Patients with Hereditary Transthyretin Amyloidosis …………… Yu RZ, Collins JW, Hall S, Ackermann EJ, Geary RS, Monia BP, Henry SP, Wang Y. Nucleic Acid Ther. 2020 Apr 15. PubMed