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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Journal ArticleDOI
Coronavirus RNA Proofreading: Molecular Basis and Therapeutic Targeting.
Fran Robson,Khadija Shahed Khan,Thi Khanh Le,Clément Paris,Sinem Demirbag,Peter Barfuss,Palma Rocchi,Wai-Lung Ng +7 more
TL;DR: The molecular basis of the CoV proofreading complex is reviewed and its potential as a drug target is evaluated and existing nucleoside analogues and novel genomic techniques are considered as potential anti-CoV therapeutics that could be used individually or in combination to target the proofreading mechanism.
Journal ArticleDOI
DNA analogues:from supramolecular principles to biological properties
TL;DR: This review focuses on recent advances in the de novo design of nucleoside analogues with improved binding properties and tries to summarize scope and limitations of this design principle.
Journal ArticleDOI
Antisense therapy in oncology: new hope for an old idea?
TL;DR: Treatment ideas in this field are summarised, clinical trials that are being done are summarized, the potential contribution of CpG motif-mediated effects are discussed, and promising molecular targets to treat human cancer with antisense oligonucleotides are looked at.
Journal ArticleDOI
Chemical modification: the key to clinical application of RNA interference?
TL;DR: RNA interference provides a potent and specific method for controlling gene expression in human cells and it might be possible to achieve this optimization using chemical modifications that improve their in vivo stability, cellular delivery, biodistribution, pharmacokinetics, potency, and specificity.
Journal ArticleDOI
Sustained dystrophin expression induced by peptide-conjugated morpholino oligomers in the muscles of mdx mice.
Natee Jearawiriyapaisarn,Hong M. Moulton,Brian Buckley,Jennifer Roberts,Peter Sazani,Suthat Fucharoen,Suthat Fucharoen,Patrick L. Iversen,Ryszard Kole,Ryszard Kole +9 more
TL;DR: PPMO M23D-B, designed to force skipping of stop-codon containing dystrophin exon 23, is investigated in an mdx mouse model of Duchenne muscular dystrophy, the first report of oligonucleotide-mediated exon skipping and dystrophic protein induction in the heart of treated animals.
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.
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,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.
Journal ArticleDOI
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.