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Journal Abstract Search

278 related items for PubMed ID: 34569970

  • 1. Effects of Chronic, Maximal Phosphorodiamidate Morpholino Oligomer (PMO) Dosing on Muscle Function and Dystrophin Restoration in a Mouse Model of Duchenne Muscular Dystrophy.
    Benny Klimek ME, Vila MC, Edwards K, Boehler J, Novak J, Zhang A, Van der Meulen J, Tatum K, Quinn J, Fiorillo A, Burki U, Straub V, Lu QL, Hathout Y, van Den Anker J, Partridge TA, Morales M, Hoffman E, Nagaraju K.
    J Neuromuscul Dis; 2021; 8(s2):S369-S381. PubMed ID: 34569970
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  • 2. Bubble liposomes and ultrasound exposure improve localized morpholino oligomer delivery into the skeletal muscles of dystrophic mdx mice.
    Negishi Y, Ishii Y, Shiono H, Akiyama S, Sekine S, Kojima T, Mayama S, Kikuchi T, Hamano N, Endo-Takahashi Y, Suzuki R, Maruyama K, Aramaki Y.
    Mol Pharm; 2014 Mar 03; 11(3):1053-61. PubMed ID: 24433046
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  • 3. Morpholino-induced exon skipping stimulates cell-mediated and humoral responses to dystrophin in mdx mice.
    Vila MC, Novak JS, Benny Klimek M, Li N, Morales M, Fritz AG, Edwards K, Boehler JF, Hogarth MW, Kinder TB, Zhang A, Mazala D, Fiorillo AA, Douglas B, Chen YW, van den Anker J, Lu QL, Hathout Y, Hoffman EP, Partridge TA, Nagaraju K.
    J Pathol; 2019 Jul 03; 248(3):339-351. PubMed ID: 30883742
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  • 4. Exon Skipping Therapy Using Phosphorodiamidate Morpholino Oligomers in the mdx52 Mouse Model of Duchenne Muscular Dystrophy.
    Miyatake S, Mizobe Y, Takizawa H, Hara Y, Yokota T, Takeda S, Aoki Y.
    Methods Mol Biol; 2018 Jul 03; 1687():123-141. PubMed ID: 29067660
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  • 8. Hexose enhances oligonucleotide delivery and exon skipping in dystrophin-deficient mdx mice.
    Han G, Gu B, Cao L, Gao X, Wang Q, Seow Y, Zhang N, Wood MJ, Yin H.
    Nat Commun; 2016 Mar 11; 7():10981. PubMed ID: 26964641
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  • 10. Exons 45-55 Skipping Using Mutation-Tailored Cocktails of Antisense Morpholinos in the DMD Gene.
    Echigoya Y, Lim KRQ, Melo D, Bao B, Trieu N, Mizobe Y, Maruyama R, Mamchaoui K, Tanihata J, Aoki Y, Takeda S, Mouly V, Duddy W, Yokota T.
    Mol Ther; 2019 Nov 06; 27(11):2005-2017. PubMed ID: 31416775
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  • 12. PMO Delivery System Using Bubble Liposomes and Ultrasound Exposure for Duchenne Muscular Dystrophy Treatment.
    Negishi Y, Ishii Y, Nirasawa K, Sasaki E, Endo-Takahashi Y, Suzuki R, Maruyama K.
    Methods Mol Biol; 2018 Nov 06; 1687():185-192. PubMed ID: 29067664
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  • 13. Enhanced exon skipping and prolonged dystrophin restoration achieved by TfR1-targeted delivery of antisense oligonucleotide using FORCE conjugation in mdx mice.
    Desjardins CA, Yao M, Hall J, O'Donnell E, Venkatesan R, Spring S, Wen A, Hsia N, Shen P, Russo R, Lan B, Picariello T, Tang K, Weeden T, Zanotti S, Subramanian R, Ibraghimov-Beskrovnaya O.
    Nucleic Acids Res; 2022 Nov 11; 50(20):11401-11414. PubMed ID: 35944903
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  • 14. Systemic Delivery of Morpholinos to Skip Multiple Exons in a Dog Model of Duchenne Muscular Dystrophy.
    Maruyama R, Echigoya Y, Caluseriu O, Aoki Y, Takeda S, Yokota T.
    Methods Mol Biol; 2017 Nov 11; 1565():201-213. PubMed ID: 28364245
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  • 17. 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 01; 25(11):2561-2572. PubMed ID: 28865998
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  • 18. Efficacy of Multi-exon Skipping Treatment in Duchenne Muscular Dystrophy Dog Model Neonates.
    Lim KRQ, Echigoya Y, Nagata T, Kuraoka M, Kobayashi M, Aoki Y, Partridge T, Maruyama R, Takeda S, Yokota T.
    Mol Ther; 2019 Jan 02; 27(1):76-86. PubMed ID: 30448197
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  • 19. Long-term rescue of dystrophin expression and improvement in muscle pathology and function in dystrophic mdx mice by peptide-conjugated morpholino.
    Wu B, Lu P, Cloer C, Shaban M, Grewal S, Milazi S, Shah SN, Moulton HM, Lu QL.
    Am J Pathol; 2012 Aug 02; 181(2):392-400. PubMed ID: 22683468
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  • 20. Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study.
    Cirak S, Arechavala-Gomeza V, Guglieri M, Feng L, Torelli S, Anthony K, Abbs S, Garralda ME, Bourke J, Wells DJ, Dickson G, Wood MJ, Wilton SD, Straub V, Kole R, Shrewsbury SB, Sewry C, Morgan JE, Bushby K, Muntoni F.
    Lancet; 2011 Aug 13; 378(9791):595-605. PubMed ID: 21784508
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