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Targeted exon skipping to address "leaky" mutations in the dystrophin gene.
Mol Ther Nucleic Acids. 2012 Oct 16;1(10):e48. doi: 10.1038/mtna.2012.40.
2
Morpholino Oligomer-Induced Dystrophin Isoforms to Map the Functional Domains in the Dystrophin Protein.
Mol Ther Nucleic Acids. 2020 Aug 21;22:263-272. doi: 10.1016/j.omtn.2020.08.019. eCollection 2020 Dec 4.
3
Dystrophin quantification and clinical correlations in Becker muscular dystrophy: implications for clinical trials.
Brain. 2011 Dec;134(Pt 12):3547-59. doi: 10.1093/brain/awr291. Epub 2011 Nov 18.
5
Targeted exon skipping to correct exon duplications in the dystrophin gene.
Mol Ther Nucleic Acids. 2014 Mar 18;3(3):e155. doi: 10.1038/mtna.2014.8.
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Antisense suppression of donor splice site mutations in the dystrophin gene transcript.
Mol Genet Genomic Med. 2013 Sep;1(3):162-73. doi: 10.1002/mgg3.19. Epub 2013 Jun 13.
8
Single Exon Skipping Can Address a Multi-Exon Duplication in the Dystrophin Gene.
Int J Mol Sci. 2020 Jun 25;21(12):4511. doi: 10.3390/ijms21124511.
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Exons 45-55 Skipping Using Mutation-Tailored Cocktails of Antisense Morpholinos in the DMD Gene.
Mol Ther. 2019 Nov 6;27(11):2005-2017. doi: 10.1016/j.ymthe.2019.07.012. Epub 2019 Jul 26.

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1
Efficacy of exon-skipping therapy for DMD cardiomyopathy with mutations in actin binding domain 1.
Mol Ther Nucleic Acids. 2023 Oct 19;34:102060. doi: 10.1016/j.omtn.2023.102060. eCollection 2023 Dec 12.
2
Systematic Approach to Developing Splice Modulating Antisense Oligonucleotides.
Int J Mol Sci. 2019 Oct 11;20(20):5030. doi: 10.3390/ijms20205030.
6
Functional correction of dystrophin actin binding domain mutations by genome editing.
JCI Insight. 2017 Sep 21;2(18). doi: 10.1172/jci.insight.95918.
9
Deletion of Dystrophin In-Frame Exon 5 Leads to a Severe Phenotype: Guidance for Exon Skipping Strategies.
PLoS One. 2016 Jan 8;11(1):e0145620. doi: 10.1371/journal.pone.0145620. eCollection 2016.
10
Antisense oligonucleotide induction of progerin in human myogenic cells.
PLoS One. 2014 Jun 3;9(6):e98306. doi: 10.1371/journal.pone.0098306. eCollection 2014.

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2
Systemic administration of PRO051 in Duchenne's muscular dystrophy.
N Engl J Med. 2011 Apr 21;364(16):1513-22. doi: 10.1056/NEJMoa1011367. Epub 2011 Mar 23.
3
Splice modification to restore functional dystrophin synthesis in Duchenne muscular dystrophy.
Curr Pharm Des. 2010;16(8):988-1001. doi: 10.2174/138161210790883480.
5
Sustained dystrophin expression induced by peptide-conjugated morpholino oligomers in the muscles of mdx mice.
Mol Ther. 2008 Sep;16(9):1624-9. doi: 10.1038/mt.2008.120. Epub 2008 Jun 10.
6
Local dystrophin restoration with antisense oligonucleotide PRO051.
N Engl J Med. 2007 Dec 27;357(26):2677-86. doi: 10.1056/NEJMoa073108.
8
Cell-penetrating-peptide-based delivery of oligonucleotides: an overview.
Biochem Soc Trans. 2007 Aug;35(Pt 4):775-9. doi: 10.1042/BST0350775.
10
Antisense oligonucleotide-induced exon skipping across the human dystrophin gene transcript.
Mol Ther. 2007 Jul;15(7):1288-96. doi: 10.1038/sj.mt.6300095. Epub 2007 Feb 6.

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