Therapy with 2'-O-Me Phosphorothioate Antisense Oligonucleotides Causes Reversible Proteinuria by Inhibiting Renal Protein Reabsorption.

Autor: Janssen MJ; Department of Pharmacology and Toxicology, Radboud University Medical Center, Radboud Institute for Molecular Life Sciences, Nijmegen, the Netherlands; Division of Pharmacology, Utrecht Institute for Pharmaceutical Sciences, Utrecht, the Netherlands. Electronic address: m.j.janssen1@uu.nl., Nieskens TTG; Department of Pharmacology and Toxicology, Radboud University Medical Center, Radboud Institute for Molecular Life Sciences, Nijmegen, the Netherlands., Steevels TAM; BioMarin Nederland B.V., Leiden, the Netherlands., Caetano-Pinto P; Department of Pharmacology and Toxicology, Radboud University Medical Center, Radboud Institute for Molecular Life Sciences, Nijmegen, the Netherlands; Division of Pharmacology, Utrecht Institute for Pharmaceutical Sciences, Utrecht, the Netherlands., den Braanker D; Department of Pharmacology and Toxicology, Radboud University Medical Center, Radboud Institute for Molecular Life Sciences, Nijmegen, the Netherlands., Mulder M; BioMarin Nederland B.V., Leiden, the Netherlands., Ponstein Y; BioMarin Nederland B.V., Leiden, the Netherlands., Jones S; BioMarin UK, London, UK., Masereeuw R; Department of Pharmacology and Toxicology, Radboud University Medical Center, Radboud Institute for Molecular Life Sciences, Nijmegen, the Netherlands; Division of Pharmacology, Utrecht Institute for Pharmaceutical Sciences, Utrecht, the Netherlands., den Besten C; BioMarin Nederland B.V., Leiden, the Netherlands., Wilmer MJ; Department of Pharmacology and Toxicology, Radboud University Medical Center, Radboud Institute for Molecular Life Sciences, Nijmegen, the Netherlands.

Publikováno v: Molecular therapy. Nucleic acids [Mol Ther Nucleic Acids] 2019 Dec 06; Vol. 18, pp. 298-307. Date of Electronic Publication: 2019 Sep 06.