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Peptide-conjugate antisense based splice-correction for Duchenne muscular dystrophy and other neuromuscular diseases
https://tokushima-u.repo.nii.ac.jp/records/2008029
https://tokushima-u.repo.nii.ac.jp/records/2008029d3faf240-190d-4532-9118-7ace35bf4b17
| 名前 / ファイル | ライセンス | アクション |
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ebiom_45_630.pdf (1.49 MB)
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| Item type | 文献 / Documents(1) | |||||||||||||||||||||||||||
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| 公開日 | 2020-12-17 | |||||||||||||||||||||||||||
| アクセス権 | ||||||||||||||||||||||||||||
| アクセス権 | open access | |||||||||||||||||||||||||||
| 資源タイプ | ||||||||||||||||||||||||||||
| 資源タイプ識別子 | http://purl.org/coar/resource_type/c_6501 | |||||||||||||||||||||||||||
| 資源タイプ | journal article | |||||||||||||||||||||||||||
| 出版社版DOI | ||||||||||||||||||||||||||||
| 関連識別子 | https://doi.org/10.1016/j.ebiom.2019.06.036 | |||||||||||||||||||||||||||
| 関連名称 | 10.1016/j.ebiom.2019.06.036 | |||||||||||||||||||||||||||
| 出版タイプ | ||||||||||||||||||||||||||||
| 出版タイプ | VoR | |||||||||||||||||||||||||||
| 出版タイプResource | http://purl.org/coar/version/c_970fb48d4fbd8a85 | |||||||||||||||||||||||||||
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| タイトル | Peptide-conjugate antisense based splice-correction for Duchenne muscular dystrophy and other neuromuscular diseases | |||||||||||||||||||||||||||
| 著者 |
Tsoumpra, Maria K.
× Tsoumpra, Maria K.
× 福本, 誠二× 松本, 俊夫
WEKO
1591
× 武田, 伸一
× Wood, Matthew J.A.
× 青木, 吉嗣
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| 抄録 | ||||||||||||||||||||||||||||
| 内容記述 | Duchenne muscular dystrophy (DMD) is an X-linked disorder characterized by progressive muscle degeneration, caused by the absence of dystrophin. Exon skipping by antisense oligonucleotides (ASOs) has recently gained recognition as therapeutic approach in DMD. Conjugation of a peptide to the phosphorodiamidate morpholino backbone (PMO) of ASOs generated the peptide-conjugated PMOs (PPMOs) that exhibit a dramatically improved pharmacokinetic profile. When tested in animal models, PPMOs demonstrate effective exon skipping in target muscles and prolonged duration of dystrophin restoration after a treatment regime. Herein we summarize the main pathophysiological features of DMD and the emergence of PPMOs as promising exon skipping agents aiming to rescue defective gene expression in DMD and other neuromuscular diseases. The listed PPMO laboratory findings correspond to latest trends in the field and highlight the obstacles that must be overcome prior to translating the animal-based research into clinical trials tailored to the needs of patients suffering from neuromuscular diseases. | |||||||||||||||||||||||||||
| 書誌情報 |
en : EBioMedicine 巻 45, p. 630-645, 発行日 2019-06-27 |
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| 収録物識別子タイプ | ISSN | |||||||||||||||||||||||||||
| 収録物識別子 | 23523964 | |||||||||||||||||||||||||||
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| 出版者 | Elsevier | |||||||||||||||||||||||||||
| 権利情報 | ||||||||||||||||||||||||||||
| 権利情報 | This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/). | |||||||||||||||||||||||||||
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| 識別子 | 361179 | |||||||||||||||||||||||||||
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| 言語 | eng | |||||||||||||||||||||||||||
