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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Peptide nucleic acids as epigenetic inhibitors of HIV-1
TL;DR: Using unmodified peptide nucleic acids (PNA) as a biomolecular tool, a potentially critical HIV gene segment within gag-polencoding gene is discovered, exemplifies the possibility that epigenic HIV inhibitors may be developed in the coming years, if emerging novel technologies permits sufficient and stable in vivo delivery of PNA or other similarly effective biomolecules.
Journal ArticleDOI
Brain Pathogenesis and Potential Therapeutic Strategies in Myotonic Dystrophy Type 1
TL;DR: In this paper, the authors summarize the current advances in the pathogenesis and pathology of central nervous system (CNS) deficits in myotonic dystrophy type 1 (DM1), and intervention measures currently being investigated are also highlighted, aiming to promote novel and cutting-edge therapeutic investigations.
Journal ArticleDOI
Bifunctional small molecule-oligonucleotide hybrid as microRNA inhibitor.
Umesh Bhattarai,Wei-Che Hsieh,Hao Yan,Zhi-Fo Guo,Ashif Y. Shaikh,Aria Soltani,Yabin Song,Danith H. Ly,Fu-Sen Liang,Fu-Sen Liang +9 more
TL;DR: This work shows that antisense oligonucleotides (ASOs), including morpholinos and γPNAs, could be readily used as the RNA recognition unit to generate bifunctional small molecule-oligonucleotide hybrids as miRNA inhibitors, and revealed that the potency of these hybrids is mainly determined by the RNA binding of the targeting ASO molecules.
Journal ArticleDOI
Antisense oligonucleotides modulate dopa decarboxylase function in aromatic l-amino acid decarboxylase deficiency.
TL;DR: It is demonstrated that antisense oligonucleotides (ASOs) were able to restore the normal mRNA splicing and increase the level of DDC protein, as well as its downstream product serotonin, in lymphoblastoid cells derived from the patient with A ADCD, suggesting that these ASOs might represent a feasible alternative strategy for gene therapy of AADCD in patients with the common c.714+4A>T mutation.
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Novel Dioxane and Morpholino Nucleotide Analogues: Syntheses and RNA-Hybridization Properties.
TL;DR: A novel class of nucleotide analogues with a dioxane ring as central scaffold has been developed, and the final thymidine analogues were synthesized with common functionalities for the automated oligonucleotide synthesis.
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.
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.