The Pharmacokinetics of 2′-O-Methyl Phosphorothioate Antisense Oligonucleotides: Experiences from Developing Exon Skipping Therapies for Duchenne Muscular Dystrophy

2019 ◽  
Vol 29 (6) ◽  
pp. 305-322 ◽  
Author(s):  
Sieto Bosgra ◽  
Jessica Sipkens ◽  
Sjef de Kimpe ◽  
Cathaline den Besten ◽  
Nicole Datson ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Antisense Oligonucleotides ◽  
Exon Skipping

In vivocomparison of 2′-O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping

2009 ◽  
Vol 11 (3) ◽  
pp. 257-266 ◽  
Author(s):  
Hans A. Heemskerk ◽  
Christa L. de Winter ◽  
Sjef J. de Kimpe ◽  
Petra van Kuik-Romeijn ◽  
Niki Heuvelmans ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Antisense Oligonucleotides ◽  
Exon Skipping

scholarly journals Novel Cationic Carotenoid Lipids as Delivery Vectors of Antisense Oligonucleotides for Exon Skipping in Duchenne Muscular Dystrophy

Molecules ◽  
2012 ◽  
Vol 17 (2) ◽  
pp. 1138-1148 ◽  
Author(s):  
Linda J. Popplewell ◽  
Aseel Abu-Dayya ◽  
Tushar Khanna ◽  
Marcella Flinterman ◽  
Nada Abdul Khalique ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Antisense Oligonucleotides ◽  
Exon Skipping

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

2018 ◽  
Vol 26 (1) ◽  
pp. 132-147 ◽  
Author(s):  
Silvana M.G. Jirka ◽  
Peter A.C. ’t Hoen ◽  
Valeriano Diaz Parillas ◽  
Christa L. Tanganyika-de Winter ◽  
Ruurd C. Verheul ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Mouse Model ◽  
Antisense Oligonucleotides ◽  
Cyclic Peptides ◽  
Exon Skipping

scholarly journals New Approach for Antisense Oligonucleotide-Mediated Exon Skipping in Duchenne Muscular Dystrophy

2012 ◽  
Vol 16 (4) ◽  
pp. 521-526
Author(s):  
Yoshitsugu Aoki ◽  
◽  
Tetsuya Nagata ◽  
Shin’ichi Takeda
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Antisense Oligonucleotide ◽  
Antisense Oligonucleotides ◽  
Exon Skipping ◽  
New Approach ◽  
Reading Frame ◽  
Promising Therapeutic Approach ◽  
Dmd Gene ◽  
Dystrophin Protein

Duchenne Muscular Dystrophy (DMD) is a lethalmuscle disorder characterized by mutations in the DMD gene. These mutations primarily disrupt the reading frame, resulting in the absence of functional dystrophin protein. Exon skipping, which involves the use of antisense oligonucleotides is a promising therapeutic approach for DMD, and clinical trials on exon skipping are currently underway in DMD patients. Recently, stable and less-toxic antisense oligonucleotides with higher efficacy have been developed in mouse and dog models of DMD. This review highlights a new approach for antisense oligonucleotide-based therapeutics for DMD, particularly for exon skipping-based methods.

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