Aartsma-Rus A - Search Results

1

Current status of pharmaceutical and genetic therapeutic approaches to treat DMD.

Pichavant C, Aartsma-Rus A, Clemens PR, Davies KE, Dickson G, Takeda S, Wilton SD, Wolff JA, Wooddell CI, Xiao X, Tremblay JP. Pichavant C, et al. Mol Ther. 2011 May;19(5):830-40. doi: 10.1038/mt.2011.59. Epub 2011 Apr 5. Mol Ther. 2011. PMID: 21468001 Free PMC article. Review.

2

Comparative analysis of antisense oligonucleotide analogs for targeted DMD exon 46 skipping in muscle cells.

Aartsma-Rus A, Kaman WE, Bremmer-Bout M, Janson AA, den Dunnen JT, van Ommen GJ, van Deutekom JC. Aartsma-Rus A, et al. Gene Ther. 2004 Sep;11(18):1391-8. doi: 10.1038/sj.gt.3302313. Gene Ther. 2004. PMID: 15229633

3

Targeted exon skipping in transgenic hDMD mice: A model for direct preclinical screening of human-specific antisense oligonucleotides.

Bremmer-Bout M, Aartsma-Rus A, de Meijer EJ, Kaman WE, Janson AA, Vossen RH, van Ommen GJ, den Dunnen JT, van Deutekom JC. Bremmer-Bout M, et al. Mol Ther. 2004 Aug;10(2):232-40. doi: 10.1016/j.ymthe.2004.05.031. Mol Ther. 2004. PMID: 15294170 Free article.

4

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.

5

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.

6

Comparative analysis of antisense oligonucleotide sequences targeting exon 53 of the human DMD gene: Implications for future clinical trials.

Popplewell LJ, Adkin C, Arechavala-Gomeza V, Aartsma-Rus A, de Winter CL, Wilton SD, Morgan JE, Muntoni F, Graham IR, Dickson G. Popplewell LJ, et al. Neuromuscul Disord. 2010 Feb;20(2):102-10. doi: 10.1016/j.nmd.2009.10.013. Epub 2010 Jan 15. Neuromuscul Disord. 2010. PMID: 20079639

7

Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases.

Tremblay JP, Xiao X, Aartsma-Rus A, Barbas C, Blau HM, Bogdanove AJ, Boycott K, Braun S, Breakefield XO, Bueren JA, Buschmann M, Byrne BJ, Calos M, Cathomen T, Chamberlain J, Chuah M, Cornetta K, Davies KE, Dickson JG, Duchateau P, Flotte TR, Gaudet D, Gersbach CA, Gilbert R, Glorioso J, Herzog RW, High KA, Huang W, Huard J, Joung JK, Liu D, Liu D, Lochmüller H, Lustig L, Martens J, Massie B, Mavilio F, Mendell JR, Nathwani A, Ponder K, Porteus M, Puymirat J, Samulski J, Takeda S, Thrasher A, VandenDriessche T, Wei Y, Wilson JM, Wilton SD, Wolfe JH, Gao G. Tremblay JP, et al. Mol Ther. 2013 Feb;21(2):266-8. doi: 10.1038/mt.2013.4. Mol Ther. 2013. PMID: 23369965 Free PMC article. No abstract available.

8

Development of a web course on gene therapy by the international consortium of gene therapy.

Tremblay JP, Aartsma-Rus A, Bogdanove A, Ferreira MB, Bueren J, Huard J. Tremblay JP, et al. Mol Ther. 2014 Mar;22(3):482. doi: 10.1038/mt.2014.11. Mol Ther. 2014. PMID: 24584076 Free PMC article. No abstract available.

9

The Pathogenesis and Therapy of Muscular Dystrophies.

Guiraud S, Aartsma-Rus A, Vieira NM, Davies KE, van Ommen GJ, Kunkel LM. Guiraud S, et al. Annu Rev Genomics Hum Genet. 2015;16:281-308. doi: 10.1146/annurev-genom-090314-025003. Epub 2015 Jun 4. Annu Rev Genomics Hum Genet. 2015. PMID: 26048046 Review.

10

Cyclic Peptides to Improve Delivery and Exon Skipping of Antisense Oligonucleotides in a Mouse Model for Duchenne Muscular Dystrophy.

Jirka SMG, 't Hoen PAC, Diaz Parillas V, Tanganyika-de Winter CL, Verheul RC, Aguilera B, de Visser PC, Aartsma-Rus AM. Jirka SMG, et al. Mol Ther. 2018 Jan 3;26(1):132-147. doi: 10.1016/j.ymthe.2017.10.004. Epub 2017 Oct 12. Mol Ther. 2018. PMID: 29103911 Free PMC article.

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