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
Citations
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Posted ContentDOI
Exon 44 skipping in Duchenne muscular dystrophy: NS-089/NCNP-02, an antisense oligonucleotide with a novel connected-sequence design
Naoki Watanabe,Yuichiro Tone,Tetsuya Nagata,Satoru Masuda,Takashi Saito,Norio Motohashi,Kazuchika Takagaki,Yoshitsugu Aoki,Shin'ichi Takeda +8 more
TL;DR: In this paper , an antisense oligonucleotide (ASO) was used for exon skipping in Duchenne muscular dystrophy (DMD) patients.
Patent
Substituted phosphorodiamidate morpholino oligomers
TL;DR: In this article, the authors provide diastereomerically pure or substantially diastomericallypure activated phosphoramidochloridate morpholino nucleosides, methods of their preparation and their use in stereospecific coupling for stereosecific synthesis of diastromatically pure PMO oligomers.
Dissertation
Classical and molecular studies enhancing the detection and control of avian leukosis virus
Journal ArticleDOI
Development of Nucleic Acid Medicines Based on Chemical Technology.
TL;DR: An overview of the development of chemistry-based technologies aimed at using nucleic acids for developing therapeutics over the past several decades, with a specific emphasis on the structural design and functionality of chemical modification strategies is provided in this article .
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.