Journal ArticleDOI
Resistance of morpholino phosphorodiamidate oligomers to enzymatic degradation.
Robert M. Hudziak,Elisabeth Barofsky,Douglas F. Barofsky,Doreen L. Weller,Sung-Ben Huang,Dwight D. Weller +5 more
TLDR
The excellent resistance of Morpholino phosphorodiamidates to enzymatic attack indicates their suitability for in vivo use.Abstract:
Oligomers possessing the Morpholino phosphorodiamidate backbone were evaluated for resistance to a variety of enzymes and biologic fluids. A 25-mer was incubated with nucleases, proteases, esterase...read more
Citations
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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 Article
Antisense Oligonucleotides: Basic Concepts and Mechanisms
Nathalie Dias,Cy A. Stein +1 more
TL;DR: This chapter discusses the use of short fragments of nucleic acid, commonly called oligonucleotides, either as therapeutic agents or as tools to study gene function.
Journal ArticleDOI
Morpholino antisense oligomers: the case for an RNase H-independent structural type.
TL;DR: 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.
Journal ArticleDOI
Morpholino oligos: making sense of antisense?
TL;DR: The evidence so far suggests that, with careful controls, morpholinos provide a relatively simple and rapid method to study gene function.
Journal ArticleDOI
Antisense oligonucleotides: the next frontier for treatment of neurological disorders
Carlo Rinaldi,Wood Mja. +1 more
TL;DR: With the rapid development of improved next-generation ASOs toward clinical application, this technology now holds the potential to have a dramatic effect on the treatment of many neurological conditions in the near future.
References
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Journal ArticleDOI
A Simple Method for Delivering Morpholino Antisense Oligos into the Cytoplasm of Cells
Michael Partridge,Alexandra Vincent,Paula Matthews,John Puma,David A. Stein,James E. Summerton +5 more
TL;DR: A simple and effective means for delivering Morpholino antisense oligos into the cytosol of cultured anchorage-dependent animal cells is reported, which indicates good sequence specificity by this structural type.
Journal ArticleDOI
Comparative evaluation of seven oligonucleotide analogues as potential antisense agents.
François Morvan,H. Porumb,G. Degols,Isabelle Lefebvre,Alain Pompon,B. S. Sproat,Bernard Rayner,Claude Malvy,B. Lebleu,Jean-Louis Imbach +9 more
TL;DR: All the modified oligonucleotides examined in the present study exhibited a sequence-nonspecific inhibitory effect on HIV-1 replication, the phosphorothioate analogues being the most active ones (ED50 < 1 microM).
Journal ArticleDOI
Oligothymidylate analogues having stereoregular, alternating methylphosphonate/phosphodiester backbones. Synthesis and physical studies.
TL;DR: The results suggest that the configuration of the methylphosphate linkage controls: 1) interaction with nucleases, 2) oligomer conformation, and 3) interactions with polynucleotides.
Journal ArticleDOI
In vitro efficacy of morpholino-modified antisense oligomers directed against tumor necrosis factor-alpha mRNA.
TL;DR: A panel of morpholino antisense oligomers (M-AS) for their ability to inhibit macrophage tumor necrosis factor-α (TNF-α) release was evaluated and phosphodiester and phosphorothioate types of oligonucleotides were compared.
Journal ArticleDOI
Stability measurements of antisense oligonucleotides by capillary gel electrophoresis
TL;DR: A simple desalting method to improve the injection efficiency and sensitivity of the method are described and the kinetic influence of a specific nuclease concentration and the antisense oligonucleotide structure on the cleavage reaction are discussed.