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Preclinical PK and PD studies on 2'-O-methyl-phosphorothioate RNA antisense oligonucleotides in the mdx mouse model.
Heemskerk H, de Winter C, van Kuik P, Heuvelmans N, Sabatelli P, Rimessi P, Braghetta P, van Ommen GJ, de Kimpe S, Ferlini A, Aartsma-Rus A, van Deutekom JC. Heemskerk H, et al. Among authors: de winter c, de kimpe s. Mol Ther. 2010 Jun;18(6):1210-7. doi: 10.1038/mt.2010.72. Epub 2010 Apr 20. Mol Ther. 2010. PMID: 20407428 Free PMC article.
Local dystrophin restoration with antisense oligonucleotide PRO051.
van Deutekom JC, Janson AA, Ginjaar IB, Frankhuizen WS, Aartsma-Rus A, Bremmer-Bout M, den Dunnen JT, Koop K, van der Kooi AJ, Goemans NM, de Kimpe SJ, Ekhart PF, Venneker EH, Platenburg GJ, Verschuuren JJ, van Ommen GJ. van Deutekom JC, et al. Among authors: de kimpe sj. N Engl J Med. 2007 Dec 27;357(26):2677-86. doi: 10.1056/NEJMoa073108. N Engl J Med. 2007. PMID: 18160687 Free article. Clinical Trial.
38 results