Journal ArticleDOI
Morpholino antisense oligomers: the case for an RNase H-independent structural type.
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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.About:
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.read more
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2'-O,4'-C-ethylene-bridged nucleic acids (ENA): highly nuclease-resistant and thermodynamically stable oligonucleotides for antisense drug.
Koji Morita,Chikako Hasegawa,Kaneko Masakatsu,Shinya Tsutsumi,Junko Sone,Tomio Ishikawa,Takeshi Imanishi,Makoto Koizumi +7 more
TL;DR: The ethylene-bridged nucleic acids (ENA) showed a high binding affinity for the complementary RNA strand (DeltaT(m)=+5.2 degrees C/modification) and were more nuclease-resistant than natural DNA and BNA/LNA.
Journal ArticleDOI
Antisense oligonucleotide-induced exon skipping across the human dystrophin gene transcript.
TL;DR: A panel of AOs designed to induce skipping of every exon within the human dystrophin gene transcript, with the exception of the first and last exons are described.
Journal ArticleDOI
Morpholino antisense oligonucleotides: tools for investigating vertebrate development.
David R. Corey,John M. Abrams +1 more
TL;DR: Antisense oligonucleotides provide a promising approach to investigating gene function in vivo, but their ability to offer unambiguous insights into phenotypes has been debated.
Journal ArticleDOI
Modulation of gene expression by antisense and antigene oligodeoxynucleotides and small interfering RNA.
TL;DR: The principles, strategies and delivery consideration of these nucleic acids are reviewed, and the ways to overcome the biological barriers are discussed so that therapeutic concentrations at their target sites can be maintained for a desired period.
Journal ArticleDOI
Dystrophin expression in the mdx mouse after localised and systemic administration of a morpholino antisense oligonucleotide
TL;DR: The potential of transcript manipulation to overcome disease‐causing mutations is investigated, using antisense oligonucleotides to induce specific exon removal during processing of the dystrophin primary transcript and thereby by‐pass protein‐truncating mutations.
References
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Journal ArticleDOI
Cpg motifs in bacterial dna trigger direct b-cell activation
Arthur M. Krieg,Ae-Kyung Yi,Sara Matson,Thomas J. Waldschmidt,Gail A. Bishop,Gail A. Bishop,Rebecca M. Teasdale,Gary A. Koretzky,Dennis M. Klinman +8 more
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.
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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.
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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,Peter O'Hare +1 more
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.
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Evaluation of 2'-modified oligonucleotides containing 2'-deoxy gaps as antisense inhibitors of gene expression
Brett P. Monia,Elena A. Lesnik,Carolyn Gonzalez,Walt F. Lima,Daniel Peter Claude Mcgee,Charles J. Guinosso,Andrew Mamoro Kawasaki,Phillip Dan Cook,Susan M. Freier +8 more
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.