Structural modifications of antisense oligonucleotides

Non-Nucleosidic Analogues of Polyaminonucleosides and Their Influence on Thermodynamic Properties of Derived Oligonucleotides

“…The combination of nucleotides with aminoalkyl chains greatly enhances the variety of possible structures as well as their potential application [24]. Thus, oligonucleotides possessing cationic functionalities in addition to the anionic phosphate backbone have been shown to exhibit promising properties [25][26][27][28]. Polyamines, putrescine, spermine and spermidine, are involved in the regulation of gene function [29,30].…”

Stepwise synthesis of RNA conjugates carrying peptide sequences for RNA interference studies

“…Most of the improvements in the design of nucleic acid derivatives aim to enhance stability to nuclease and improve cell uptake without affecting the hybridisation properties, which are vital for the efficient inhibitory properties of oligonucleotides. A large number of nucleic acid derivatives have been developed [5,7]. The conjugation of lipids to oligonucleotides, such as cholesterol [8,9], produces molecules with improved inhibitory properties [10,11].…”

Dendrimer delivery of an anti-VEGF oligonucleotide into the eye: a long-term study into inhibition of laser-induced CNV, distribution, uptake and toxicity

“…These therapies include gene transfer and endogenous expression of recombinant proteins, 5,6 use of pegalated aptamers to bind and inhibit activity of excess VEGF 7 and oligonucleotide gene therapy. [8][9][10] Previously, we have reported on the discovery and use of a sense oligonucleotide (ODN-1) that is complimentary to a section of sequence in the 5 0 untranslated region of VEGF, for the inhibition of VEGF expression both in vitro and in vivo. We subsequently tested the efficacy of ODN-1 in reducing the extent of laser photocoagulation induced choroidal neovascularisation (CNV) in a rodent model.…”