Journal ArticleDOI
Morpholino antisense oligomers: the case for an RNase H-independent structural type.
Reads0
Chats0
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
More filters
Journal ArticleDOI
In vivo Bioavailability and Pharmacokinetics of a c-MYC Antisense Phosphorodiamidate Morpholino Oligomer, AVI-4126, in Solid Tumors
Gayathri R. Devi,Tomasz M. Beer,Christopher L. Corless,Vikram Arora,Doreen L. Weller,Patrick L. Iversen +5 more
TL;DR: These studies show PMO bioavailability in tumor tissue and establish the feasibility of using PMO targeting specific genes in human cancer clinical trials.
Journal ArticleDOI
Influence of diverse chemical modifications on the ADME characteristics and toxicology of antisense oligonucleotides
Mehrdad Dirin,Johannes Winkler +1 more
TL;DR: There is currently no efficient alternative to PS, although doubts about long-term toxicity of this class still remain, and improved structural modifications or galenic formulations are deemed necessary to create entities with higher efficiency and lower toxicity.
Journal ArticleDOI
Chimeric RNA/ethylene-bridged nucleic acids promote dystrophin expression in myocytes of duchenne muscular dystrophy by inducing skipping of the nonsense mutation-encoding exon.
Agus Surono,Tran van Khanh,Yasuhiro Takeshima,Hiroko Wada,Mariko Yagi,Miho Takagi,Makoto Koizumi,Masafumi Matsuo +7 more
TL;DR: The results suggest that an RNA/ENA chimera can be used to express dystrophin in DMD, and among the specific oligonucleotides tested, an 18-mer RNA/ANA chimera was found to have the strongest activity, inducing exon 41 skipping in nearly 90% of dystrophic mRNA.
Journal ArticleDOI
Antisense morpholino-oligomers directed against the 5' end of the genome inhibit coronavirus proliferation and growth.
Benjamin W. Neuman,David A. Stein,Andrew D. Kroeker,Amy D. Paulino,Hong M. Moulton,Patrick L. Iversen,Michael J. Buchmeier +6 more
TL;DR: Conjugation of a peptide related to the human immunodeficiency virus type 1 Tat represents a novel method for delivery of antisense morpholino-oligomers that has therapeutic potential for control of coronavirus infection.
Journal ArticleDOI
Inhibition of Influenza Virus Infection in Human Airway Cell Cultures by an Antisense Peptide-Conjugated Morpholino Oligomer Targeting the Hemagglutinin-Activating Protease TMPRSS2
TL;DR: The data indicate that PPMO provide a useful reagent for investigating HA-activating proteases and may represent a promising strategy for the development of novel therapeutics to address influenza infections.
References
More filters
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.