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Journal ArticleDOI

Morpholino antisense oligomers: the case for an RNase H-independent structural type.

James Summerton
- 10 Dec 1999 - 
- Vol. 1489, Iss: 1, pp 141-158
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TLDR
In cell-free and cultured-cell systems where one wishes to block the translation of a messenger RNA coding for a normal protein, RNase H-independent morpholino antisense oligos provide complete resistance to nucleases, generally good targeting predictability, generally high in-cell efficacy, excellent sequence specificity, and very preliminary results suggest they may exhibit little non-antisense activity.
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This article is published in Biochimica et Biophysica Acta.The article was published on 1999-12-10. It has received 689 citations till now. The article focuses on the topics: RNase P & RNase H.

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Citations
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Journal ArticleDOI

Antisense applications for biological control.

TL;DR: Modifications to ASRs to enhance stability, improve targeting, and incremental improvements in delivery vehicles continue to make ASRs attractive as molecular therapeutics, but their advance toward the bedside has been agonizingly slow.
Journal ArticleDOI

Subcellular Localization of LGN During Mitosis: Evidence for Its Cortical Localization In Mitotic Cell Culture Systems And Its Requirement For Normal Cell Cycle Progression

TL;DR: It is demonstrated here that, like Drosophila Pins, LGN can exhibit enriched localization at the cell cortex, depending on the cell cycle and the culture system used, and that interfering with LGN function in cultured cell lines causes early disruption to cell cycle progression.
Journal ArticleDOI

Molecular identity of the late sodium current in adult dog cardiomyocytes identified by Nav1.5 antisense inhibition

TL;DR: It is concluded that in adult dog heart Na(v)1.5 sodium channels have a "functional half-life" of approximately 35 h (0.69tau) and make a major contribution to I(NaL), which is a major component of the action potential plateau in human and canine myocardium.
Patent

Multiple exon skipping compositions for dmd

Peter Sazani, +1 more
TL;DR: In this paper, the authors present a method to induce exon skipping in the human dystrophin gene to treat muscular dystrophy, provided that antisense molecules are capable of binding to a selected target site in the target site.
Journal ArticleDOI

The RNA-binding protein fragile X-related 1 regulates somite formation in Xenopus laevis.

TL;DR: In this paper, the authors have isolated cDNAs for Xenopus Fxr1 and found that two specific splice variants are conserved in evolution and showed that the absence of the long muscle-specific xFxr 1p isoform during early somite formation had highly musclespecific effects, normal MyoD expression being disrupted, somitic myotomal cell rotation and segmentation being inhibited, and dermatome formation being abnormal.
References
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Journal ArticleDOI

Cpg motifs in bacterial dna trigger direct b-cell activation

TL;DR: The potent immune activation by CpG oligon nucleotides has impli-cations for the design and interpretation of studies using 'antisense' oligonucleotides and points to possible new applications as adjuvants.
Journal ArticleDOI

The third helix of the Antennapedia homeodomain translocates through biological membranes

TL;DR: It is reported here that a polypeptide of 16 amino acids in length corresponding to the third helix of the homeodomain deleted of its N-terminal glutamate is still capable of translocating through the membrane, suggesting an energy-independent mechanism of translocation not involving classical endocytosis.
Journal ArticleDOI

Morpholino antisense oligomers: design, preparation, and properties.

TL;DR: An overview of the design, preparation, and properties of Morpholino oligos, a novel antisense structural type that solves the sequence specificity problem and provides high and predictable activity in cells.
Journal ArticleDOI

Intercellular trafficking and protein delivery by a herpesvirus structural protein.

Gillian Elliott, +1 more
- 24 Jan 1997 - 
TL;DR: It is shown that the HSV-1 structural protein VP22 has the remarkable property of intercellular transport, which is so efficient that following expression in a subpopulation the protein spreads to every cell in a monolayer, where it concentrates in the nucleus and binds chromatin.
Journal ArticleDOI

Evaluation of 2'-modified oligonucleotides containing 2'-deoxy gaps as antisense inhibitors of gene expression

TL;DR: The use of a previously described 17-mer phosphorothioate for structure-function analysis of 2'-sugar modifications and the results demonstrate the importance of target affinity in the action of antisense oligonucleotides and of RNase H as a mechanism by which these compounds exert their effects.
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