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

• Main Authors: Manoe J. Janssen (Author), Tom T.G. Nieskens (Author), Tessa A.M. Steevels (Author), Pedro Caetano-Pinto (Author), Dirk den Braanker (Author), Melissa Mulder (Author), Yolanda Ponstein (Author), Shaun Jones (Author), Rosalinde Masereeuw (Author), Cathaline den Besten (Author), Martijn J. Wilmer (Author)
• Format: Book
• Published: Elsevier, 2019-12-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Antisense oligonucleotide therapy has been reported to be associated with renal injury. Here, the mechanism of reversible proteinuria was investigated by combining clinical, pre-clinical, and in vitro data. Urine samples were obtained from Duchenne muscular dystrophy (DMD) patients treated with dris...