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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PNA-based artificial nucleases as antisense and anti-miRNA oligonucleotide agents
TL;DR: The results have highlighted the best performance of nuclease B and the relevance of the PEG spacer, in particular for conjugate A, in terms of efficiency of the cleavage, suggesting that conjugates A and B also act as potential antisense and anti-miRNA agents.
Journal ArticleDOI
Splice-correcting oligonucleotides restore BTK function in X-linked agammaglobulinemia model
Burcu Bestas,Pedro Moreno,K. Emelie M. Blomberg,Dara K. Mohammad,Amer F. Saleh,Tolga Sutlu,Joel Z. Nordin,Peter Guterstam,Manuela O. Gustafsson,Shabnam Kharazi,Barbara Piątosa,Thomas C. Roberts,Mark A. Behlke,Matthew J.A. Wood,Michael J. Gait,Karin E. Lundin,Samir El Andaloussi,Robert Månsson,Anna Berglöf,Jesper Wengel,C. I. Edvard Smith +20 more
TL;DR: It is demonstrated that antisense, splice-correcting oligonucleotides targeting mutated BTK transcripts can restore BTK function and that BTK-targeting SCOs have potential as personalized medicine in patients with XLA.
Journal ArticleDOI
A protein disulfide isomerase expressed in the embryonic midline is required for left/right asymmetries
TL;DR: It is proposed that PDI-P5 is specifically involved in the production of midline-derived signals required to establish left/right asymmetry in the zebrafish embryo.
Journal ArticleDOI
Oligonucleotide-based theranostic nanoparticles in cancer therapy.
TL;DR: The advantages of theranostic applications in cancer therapy and imaging, with special attention to oligonucleotide-based therapeutics are reviewed, with particular attention to quantum dots, iron oxide nanoparticles and gold nanoparticles.
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Transmembrane sema4E guides branchiomotor axons to their targets in zebrafish.
TL;DR: Results show that Sema4E is repulsive for facial and gill motor axons and functions as a barrier for these axons within the pharyngeal arches.
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.