These tools will no longer be maintained as of December 31, 2024. Archived website can be found here. PubMed4Hh GitHub repository can be found here. Contact NLM Customer Service if you have questions.
5. Nonclinical Exon Skipping Studies with 2'-O-Methyl Phosphorothioate Antisense Oligonucleotides in mdx and mdx-utrn-/- Mice Inspired by Clinical Trial Results. van Putten M; Tanganyika-de Winter C; Bosgra S; Aartsma-Rus A Nucleic Acid Ther; 2019 Apr; 29(2):92-103. PubMed ID: 30672725 [TBL] [Abstract][Full Text] [Related]
6. 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 J Gene Med; 2009 Mar; 11(3):257-66. PubMed ID: 19140108 [TBL] [Abstract][Full Text] [Related]
7. 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 Mol Ther; 2018 Jan; 26(1):132-147. PubMed ID: 29103911 [TBL] [Abstract][Full Text] [Related]
8. Prednisolone treatment does not interfere with 2'-O-methyl phosphorothioate antisense-mediated exon skipping in Duchenne muscular dystrophy. Verhaart IE; Heemskerk H; Karnaoukh TG; Kolfschoten IG; Vroon A; van Ommen GJ; van Deutekom JC; Aartsma-Rus A Hum Gene Ther; 2012 Mar; 23(3):262-73. PubMed ID: 22017442 [TBL] [Abstract][Full Text] [Related]
9. The Use of Antisense Oligonucleotides for the Treatment of Duchenne Muscular Dystrophy. Relizani K; Goyenvalle A Methods Mol Biol; 2018; 1687():171-183. PubMed ID: 29067663 [TBL] [Abstract][Full Text] [Related]
11. Exon skipping: a first in class strategy for Duchenne muscular dystrophy. Niks EH; Aartsma-Rus A Expert Opin Biol Ther; 2017 Feb; 17(2):225-236. PubMed ID: 27936976 [TBL] [Abstract][Full Text] [Related]
12. New developments in exon skipping and splice modulation therapies for neuromuscular diseases. Touznik A; Lee JJ; Yokota T Expert Opin Biol Ther; 2014 Jun; 14(6):809-19. PubMed ID: 24620745 [TBL] [Abstract][Full Text] [Related]
13. Tips to Design Effective Splice-Switching Antisense Oligonucleotides for Exon Skipping and Exon Inclusion. Maruyama R; Yokota T Methods Mol Biol; 2018; 1828():79-90. PubMed ID: 30171536 [TBL] [Abstract][Full Text] [Related]
15. Challenges of Assessing Exon 53 Skipping of the Human Engelbeen S; O'Reilly D; Van De Vijver D; Verhaart I; van Putten M; Hariharan V; Hassler M; Khvorova A; Damha MJ; Aartsma-Rus A Nucleic Acid Ther; 2023 Dec; 33(6):348-360. PubMed ID: 38010230 [TBL] [Abstract][Full Text] [Related]
16. Exonic sequences provide better targets for antisense oligonucleotides than splice site sequences in the modulation of Duchenne muscular dystrophy splicing. Aartsma-Rus A; Houlleberghs H; van Deutekom JC; van Ommen GJ; 't Hoen PA Oligonucleotides; 2010 Apr; 20(2):69-77. PubMed ID: 20377429 [TBL] [Abstract][Full Text] [Related]
17. A prospective study in the rational design of efficient antisense oligonucleotides for exon skipping in the DMD gene. Pramono ZA; Wee KB; Wang JL; Chen YJ; Xiong QB; Lai PS; Yee WC Hum Gene Ther; 2012 Jul; 23(7):781-90. PubMed ID: 22486275 [TBL] [Abstract][Full Text] [Related]
18. Development of antisense-mediated exon skipping as a treatment for duchenne muscular dystrophy. Heemskerk H; de Winter CL; van Ommen GJ; van Deutekom JC; Aartsma-Rus A Ann N Y Acad Sci; 2009 Sep; 1175():71-9. PubMed ID: 19796079 [TBL] [Abstract][Full Text] [Related]
19. Quantitative Antisense Screening and Optimization for Exon 51 Skipping in Duchenne Muscular Dystrophy. Echigoya Y; Lim KRQ; Trieu N; Bao B; Miskew Nichols B; Vila MC; Novak JS; Hara Y; Lee J; Touznik A; Mamchaoui K; Aoki Y; Takeda S; Nagaraju K; Mouly V; Maruyama R; Duddy W; Yokota T Mol Ther; 2017 Nov; 25(11):2561-2572. PubMed ID: 28865998 [TBL] [Abstract][Full Text] [Related]
20. 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 Mol Ther; 2006 Sep; 14(3):401-7. PubMed ID: 16753346 [TBL] [Abstract][Full Text] [Related] [Next] [New Search]