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

Autor: Manoe J. Janssen, Tom T.G. Nieskens, Tessa A.M. Steevels, Pedro Caetano-Pinto, Dirk den Braanker, Melissa Mulder, Yolanda Ponstein, Shaun Jones, Rosalinde Masereeuw, Cathaline den Besten, Martijn J. Wilmer

Publikováno v: Molecular Therapy: Nucleic Acids, Vol 18, Iss , Pp 298-307 (2019)

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 m

Externí odkaz: https://doaj.org/article/b87e4abe3c8947bda573024c4f50791f

The Pharmacokinetics of 2′-O-Methyl Phosphorothioate Antisense Oligonucleotides: Experiences from Developing Exon Skipping Therapies for Duchenne Muscular Dystrophy

Autor: Nicole A. Datson, Cathaline den Besten, Judith C.T. van Deutekom, Sieto Bosgra, Sjef J. de Kimpe, Jessica A. Sipkens

Publikováno v: Nucleic Acid Therapeutics. 29:305-322

Delivery to the target site and adversities related to off-target exposure have made the road to clinical success and approval of antisense oligonucleotide (AON) therapies challenging. Various classes of AONs have distinct chemical features and pharm

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::75f7c6184e4d7580eb6c29c0db62263d
https://doi.org/10.1089/nat.2019.0805

Antisense oligonucleotide-based treatment of retinitis pigmentosa caused by USH2A exon 13 mutations

Autor: Gerard Platenburg, Jingjing Zang, Erwin van Wijk, Margo Dona, Sanne Broekman, Jiayi Miao, Cathaline den Besten, Erik de Vrieze, Hee Lam Chan, Janne J. Turunen, Hanka Venselaar, Lars Vorthoren, Kalyan Dulla, Peter Adamson, Ralph Slijkerman, Stephan C.F. Neuhauss, Silvia Albert, Wouter Beumer, Theo A. Peters, Iris Schmidt, Hester van Diepen, Ronald J.E. Pennings, Hannie Kremer, Levi Buil, Iris Schulkens

Publikováno v: Molecular Therapy, 29, 8, pp. 2441-2455
Mol Ther
Molecular Therapy, 29, 2441-2455

Mutations in USH2A are among the most common causes of syndromic and non-syndromic retinitis pigmentosa (RP). The two most recurrent mutations in USH2A, c.2299delG and c.2276G > T, both reside in exon 13. Skipping exon 13 from the USH2A transcript pr

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::0009073ca7e61b8b3ea940e0d6aa4cb9
https://hdl.handle.net/1887/3627509

Impurities in Oligonucleotide Drug Substances and Drug Products

Autor: Samantha Gao-Sheridan, Neal Sharpe, Brigitte Burm, Jeffrey W.-D. Foy, Andy Teasdale, Daniel C. Capaldi, Scott P. Henry, Cathaline den Besten, Matthias Kretschmer, Nadim Akhtar, Ben Andrews

Publikováno v: Nucleic Acid Therapeutics. 27:309-322

This white paper, which is the 10th in a series intended to address issues associated with the development of therapeutic oligonucleotides, examines the subject of product-related impurities. The authors consider chemistry and safety aspects and adva

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::44473bdb213b9944ea0a58a22446df3b
https://doi.org/10.1089/nat.2017.0691

Biophysical and physicochemical methods differentiate highly ligand-efficient human D-amino acid oxidase inhibitors

Autor: Firas Bassissi, Jennifer Venhorst, Natasja de Bruin, Chris Oostenbrink, Natalia Markova, Cathaline den Besten, Stephanie B.A. de Beer, J. Frankena, Chris G. Kruse, Maria J.P. van Dongen, Josephus H. M. Lange

Publikováno v: Lange, J H, Venhorst, J, Dongen, M J, Frankena, J, Bassissi, F, Bruin, N M, Besten, C, de Beer, S B A, Oostenbrink, B C, Markova, N & Kruse, C G 2011, ' Biophysical and physicochemical methods differentiate highly ligand-efficient human D-amino acid oxidase inhibitors ', European Journal of Medicinal Chemistry, vol. 46, no. 10, pp. 4808-4819 . https://doi.org/10.1016/j.ejmech.2011.04.023
European Journal of Medicinal Chemistry, 46(10), 4808-4819. Elsevier Masson SAS

Many early drug research efforts are too reductionist thereby not delivering key parameters such as kinetics and thermodynamics of target–ligand binding. A set of human D-Amino Acid Oxidase (DAAO) inhibitors 1–6 was applied to demonstrate the imp

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::69bb10788fb3a7a1f60acd6756d2e139
https://research.vu.nl/en/publications/d254dd15-b9b2-4a0f-9b2a-f9471647abbd