Publications

1.  Veltrop M, van Vliet L, Hulsker M, Claassens J, Brouwers C, Breukel C, van der Kaa J, Linssen MM, den Dunnen JT, Verbeek S, Aartsma-Rus A, van Putten M. A dystrophic Duchenne mouse model for testing human antisense oligonucleotides. PLoS One. (2018) Feb 21;13(2):e0193289. doi: 10.1371/journal.pone.0193289. eCollection 2018. PubMed PMID: 29466448.

2.  Aartsma-Rus A, Straub V, Hemmings R, Haas M, Schlosser-Weber G, Stoyanova-Beninska V, Mercuri E, Muntoni F, Sepodes B, Vroom E, Balabanov P. Development of Exon Skipping Therapies for Duchenne Muscular Dystrophy: A Critical Review and a Perspective on the Outstanding Issues. Nucleic Acid Ther. (2017) Oct;27(5):251-259. doi: 10.1089/nat.2017.0682. Epub 2017 Aug 10. PubMed PMID: 28796573; PubMed Central PMCID: PMC5649120.

3.  Niks EH, Aartsma-Rus A. Exon skipping: a first in class strategy for Duchenne muscular dystrophy. Expert Opin Biol Ther. (2017) Feb;17(2):225-236. doi: 10.1080/14712598.2017.1271872. Epub 2016 Dec 23. Review. PubMed PMID: 27936976. 

4.  Aartsma-Rus A, Krieg AM. FDA Approves Eteplirsen for Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga. Nucleic Acid Ther. (2017) Feb;27(1):1-3. doi: 10.1089/nat.2016.0657. Epub 2016 Dec 8. PubMed PMID: 27929755; PubMed Central PMCID: PMC5312460.

5.  Straub V, Balabanov P, Bushby K, Ensini M, Goemans N, De Luca A, Pereda A,Hemmings R, Campion G, Kaye E, Arechavala-Gomeza V, Goyenvalle A, Niks E, Veldhuizen O, Furlong P, Stoyanova-Beninska V, Wood MJ, Johnson A, Mercuri E, Muntoni F, Sepodes B, Haas M, Vroom E, Aartsma-Rus A. Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy. Lancet Neurol. (2016) Jul;15(8):882-90. doi: 10.1016/S1474-4422(16)30035-7. Review. PubMed PMID: 27302365.

6.  Gazzoli I, Pulyakhina I, Verwey NE, Ariyurek Y, Laros JF, 't Hoen PA and Aartsma-Rus A. Non-sequential and multi-step splicing of the dystrophin transcript. RNA Biol. (2015), epub Dec 15.

7.  Van Putten M, Hulsker M, Young C, Nadarajah VD, Heemskerk H, van der Weerd L, ‘t Hoen PAC, van Ommen GJB and Aartsma-Rus A. Low dystrophin levels increase survival and improve muscle pathology and function in dystrophin/utrophin double knockout mice. FASEB (2013); 27: 2484-95. 

8.  Goemans N, Tulinius M, van den Akker JT…..Aartsma-Rus A…and van Deutekom JCT. Systemic administration of PRO051 in Duchenne’s muscular dystrophy. N Engl J Med (2011); 364: 1513-22 

9.  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. Local dystrophin restoration with antisense oligonucleotide PRO051. N Engl J Med. (2007) Dec 27;357(26):2677-86. PubMed PMID: 18160687.

10. Aartsma-Rus A, Janson AA, Kaman WE, Bremmer-Bout M, den Dunnen JT, Baas F, van Ommen GJ, van Deutekom JC. Therapeutic antisense-induced exon skipping in cultured muscle cells from six different DMD patients. Hum Mol Genet. (2003) Apr15;12(8):907-14. PubMed PMID: 12668614.

All publications