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Related Experiment Video

Updated: Jun 1, 2026

Sequence-specific and Selective Recognition of Double-stranded RNAs over Single-stranded RNAs by Chemically Modified Peptide Nucleic Acids

Sequence-specific and Selective Recognition of Double-stranded RNAs over Single-stranded RNAs by Chemically Modified Peptide Nucleic Acids

Published on: September 21, 2017

PNA-based artificial nucleases as antisense and anti-miRNA oligonucleotide agents.

M Gaglione¹, G Milano, A Chambery

¹Environmental Sciences Department, SUN, 81100-Caserta, Italy.

Molecular Biosystems

|May 31, 2011

Summary

Peptide Nucleic Acid (PNA)-based nucleases show promise as gene therapy agents. Nuclease B demonstrated superior performance, highlighting PNA

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In Vivo Nanovector Delivery of a Heart-specific MicroRNA-sponge

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Last Updated: Jun 1, 2026

Sequence-specific and Selective Recognition of Double-stranded RNAs over Single-stranded RNAs by Chemically Modified Peptide Nucleic Acids

Sequence-specific and Selective Recognition of Double-stranded RNAs over Single-stranded RNAs by Chemically Modified Peptide Nucleic Acids

Published on: September 21, 2017

In Vivo Nanovector Delivery of a Heart-specific MicroRNA-sponge

In Vivo Nanovector Delivery of a Heart-specific MicroRNA-sponge

Published on: June 15, 2018

Area of Science:

* Molecular Biology
* Nucleic Acid Chemistry
* Drug Discovery

Background:

* Peptide Nucleic Acid (PNA) exhibits unique chemical, physical, and biological properties.
* PNA is recognized for its potential in diagnostics, biosensors, and gene therapeutics, particularly in antisense (AO) and anti-miRNA (AMO) strategies.
* PNA-based oligonucleotides are emerging as effective agents for AO and AMO approaches.

Purpose of the Study:

* To design, synthesize, and characterize novel PNA-based artificial nucleases.
* To evaluate the nuclease activity of two distinct PNA-PEG conjugates (A and B) with different cleavage sites (HGG·Cu and DETA).
* To assess the potential of these PNA-based nucleases as AO and AMO agents.

Main Methods:

* Synthesis and characterization of PEG-PNA-PEG constructs conjugated to HGG·Cu (A) and DETA (B).
* Qualitative analysis using MALDI-TOF mass spectrometry.
* Quantitative nuclease activity assays using High-Throughput Screening (HTS) on an RNA-3'-FAM target sequence.

Main Results:

* Both PNA-based nucleases (A and B) demonstrated nuclease activity against the RNA target.
* Nuclease B exhibited superior performance compared to nuclease A.
* The presence of a Polyethylene Glycol (PEG) spacer was found to be relevant for cleavage efficiency, especially in conjugate A.

Conclusions:

* PNA-based nucleases, particularly conjugate B, show significant potential as AO and AMO agents.
* The study underscores the importance of the PEG spacer in enhancing the efficiency of PNA-based nucleases.
* These findings suggest a promising avenue for developing novel gene-modulating therapeutics using PNA technology.