Aartsma-Rus A - Search Results

1

Association Study of Exon Variants in the NF-κB and TGFβ Pathways Identifies CD40 as a Modifier of Duchenne Muscular Dystrophy.

Bello L, Flanigan KM, Weiss RB; United Dystrophinopathy Project; Spitali P, Aartsma-Rus A, Muntoni F, Zaharieva I, Ferlini A, Mercuri E, Tuffery-Giraud S, Claustres M, Straub V, Lochmüller H, Barp A, Vianello S, Pegoraro E, Punetha J, Gordish-Dressman H, Giri M, McDonald CM, Hoffman EP; Cooperative International Neuromuscular Research Group. Bello L, et al. Am J Hum Genet. 2016 Nov 3;99(5):1163-1171. doi: 10.1016/j.ajhg.2016.08.023. Epub 2016 Oct 13. Am J Hum Genet. 2016. PMID: 27745838 Free PMC article.

2

Targeted exon skipping as a potential gene correction therapy for Duchenne muscular dystrophy.

Aartsma-Rus A, Bremmer-Bout M, Janson AA, den Dunnen JT, van Ommen GJ, van Deutekom JC. Aartsma-Rus A, et al. Neuromuscul Disord. 2002 Oct;12 Suppl 1:S71-7. doi: 10.1016/s0960-8966(02)00086-x. Neuromuscul Disord. 2002. PMID: 12206800

3

Therapeutic antisense-induced exon skipping in cultured muscle cells from six different DMD patients.

Aartsma-Rus A, Janson AA, Kaman WE, Bremmer-Bout M, den Dunnen JT, Baas F, van Ommen GJ, van Deutekom JC. Aartsma-Rus A, et al. Hum Mol Genet. 2003 Apr 15;12(8):907-14. doi: 10.1093/hmg/ddg100. Hum Mol Genet. 2003. PMID: 12668614

4

Antisense-induced multiexon skipping for Duchenne muscular dystrophy makes more sense.

Aartsma-Rus A, Janson AA, Kaman WE, Bremmer-Bout M, van Ommen GJ, den Dunnen JT, van Deutekom JC. Aartsma-Rus A, et al. Am J Hum Genet. 2004 Jan;74(1):83-92. doi: 10.1086/381039. Epub 2003 Dec 16. Am J Hum Genet. 2004. PMID: 14681829 Free PMC article.

5

Exploring the frontiers of therapeutic exon skipping for Duchenne muscular dystrophy by double targeting within one or multiple exons.

Aartsma-Rus A, Kaman WE, Weij R, den Dunnen JT, van Ommen GJ, van Deutekom JC. Aartsma-Rus A, et al. Mol Ther. 2006 Sep;14(3):401-7. doi: 10.1016/j.ymthe.2006.02.022. Epub 2006 Jun 6. Mol Ther. 2006. PMID: 16753346 Free article.

6

Duplications in the DMD gene.

White SJ, Aartsma-Rus A, Flanigan KM, Weiss RB, Kneppers AL, Lalic T, Janson AA, Ginjaar HB, Breuning MH, den Dunnen JT. White SJ, et al. Hum Mutat. 2006 Sep;27(9):938-45. doi: 10.1002/humu.20367. Hum Mutat. 2006. PMID: 16917894

7

Antisense-induced exon skipping for duplications in Duchenne muscular dystrophy.

Aartsma-Rus A, Janson AA, van Ommen GJ, van Deutekom JC. Aartsma-Rus A, et al. BMC Med Genet. 2007 Jul 5;8:43. doi: 10.1186/1471-2350-8-43. BMC Med Genet. 2007. PMID: 17612397 Free PMC article.

8

Comparative analysis of antisense oligonucleotide sequences for targeted skipping of exon 51 during dystrophin pre-mRNA splicing in human muscle.

Arechavala-Gomeza V, Graham IR, Popplewell LJ, Adams AM, Aartsma-Rus A, Kinali M, Morgan JE, van Deutekom JC, Wilton SD, Dickson G, Muntoni F. Arechavala-Gomeza V, et al. Hum Gene Ther. 2007 Sep;18(9):798-810. doi: 10.1089/hum.2006.061. Hum Gene Ther. 2007. PMID: 17767400 Free article.

9

Assessment of the feasibility of exon 45-55 multiexon skipping for Duchenne muscular dystrophy.

van Vliet L, de Winter CL, van Deutekom JC, van Ommen GJ, Aartsma-Rus A. van Vliet L, et al. BMC Med Genet. 2008 Dec 1;9:105. doi: 10.1186/1471-2350-9-105. BMC Med Genet. 2008. PMID: 19046429 Free PMC article.

10

In vivo comparison of 2'-O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping.

Heemskerk HA, de Winter CL, de Kimpe SJ, van Kuik-Romeijn P, Heuvelmans N, Platenburg GJ, van Ommen GJ, van Deutekom JC, Aartsma-Rus A. Heemskerk HA, et al. J Gene Med. 2009 Mar;11(3):257-66. doi: 10.1002/jgm.1288. J Gene Med. 2009. PMID: 19140108

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