Shorter Phosphorodiamidate Morpholino Splice-Switching Oligonucleotides May Increase Exon-Skipping Efficacy in DMD
Yazarlar (7)
Doç. Dr. Uğur AKPULAT Kastamonu Üniversitesi, Türkiye
Haicui Wang Universität Zu Köln, Almanya
Kerstin Becker Universität Zu Köln, Almanya
Adriana Contreras Universität Zu Köln, Almanya
Terence Partridge Children's Research Institute, Children's National Medical Center, Amerika Birleşik Devletleri
James Novak Children's Research Institute, Children's National Medical Center, Amerika Birleşik Devletleri
Sebahattin Cirak Universität Zu Köln, Almanya
| Makale Türü |
Özgün Makale
(SSCI, AHCI, SCI, SCI-Exp dergilerinde yayınlanan tam makale)
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| Dergi Adı | Molecular Therapy Nucleic Acids (Q1) | ||
| Dergi ISSN | 2162-2531 Wos Dergi Scopus Dergi | ||
| Dergi Tarandığı Indeksler | SCI-Expanded | ||
| Makale Dili | İngilizce | Basım Tarihi | 12-2018 |
| Cilt / Sayı / Sayfa | 13 / 1 / 534–542 | DOI | 10.1016/j.omtn.2018.10.002 |
| Makale Linki | https://doi.org/10.1016/j.omtn.2018.10.002 | ||
| UAK Araştırma Alanları |
Tıbbi Biyoloji
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| Özet |
| Duchenne muscular dystrophy is a fatal muscle disease, caused by mutations in DMD, leading to loss of dystrophin expression. Phosphorodiamidate morpholino splice-switching oligonucleotides (PMO-SSOs) have been used to elicit the restoration of a partially functional truncated dystrophin by excluding disruptive exons from the DMD messenger. The 30-mer PMO eteplirsen (EXONDYS51) developed for exon 51 skipping is the first dystrophin-restoring, conditionally FDA-approved drug in history. Clinical trials had shown a dose-dependent variable and patchy dystrophin restoration. The main obstacle for efficient dystrophin restoration is the inadequate uptake of PMOs into skeletal muscle fibers at low doses. The excessive cost of longer PMOs has limited the utilization of higher dosing. We designed shorter 25-mer PMOs directed to the same eteplirsen-targeted region of exon 51 and compared their efficacies in … |
| Anahtar Kelimeler |
| DMD | dystrophin | eteplirsen | exon skipping | exondys51 | mdx | myopathy | phosphorodiamidate morpholino | phosphorothiorate | PMO | shorter PMO-SSOs |
Science Direct
Shorter Phosphorodiamidate Morpholino Splice-Switching Oligonucleotides May Increase Exon-Skipping Efficacy in DMD
BM Sürdürülebilir Kalkınma Amaçları
| Atıf Sayıları | |
| Web of Science | 9 |
| Scopus | 10 |
| Google Scholar | 14 |