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RNA Editing

RNA Editing

RNA editing is a post-transcriptional modification where a precursor mRNA (pre-mRNA) nucleotide sequence is changed by base insertion, deletion, or modification. The extent of RNA editing varies from a few hundred bases, in mitochondrial DNA of trypanosomes, to a just single base, in nuclear genes of mammals. Even a single base change in the pre-mRNA can convert a codon for one amino acid into the codon for another amino acid or a stop codon. This type of re-coding can significantly affect the...

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Efficient Transfection of In vitro Transcribed mRNA in Cultured Cells Using Peptide-Poloxamine Nanoparticles

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Polyamine-Modified Poly(aspartic acid) for mRNA Delivery with In Vivo Lung-Targeted Ability.

Yuyan Zhang^1,2, Pingjie Sun^1,3, Sheng Ma^1,3

¹State Key Laboratory of Polymer Science and Technology, Changchun Institute of Applied Chemistry, Chinese Academy of Sciences, Changchun 130022, China.

Biomacromolecules

|October 15, 2025

Summary

New poly(amino acid) polymers efficiently deliver mRNA to the lungs. These polyamine-aminolyzed PAsp derivatives (P-An) show promise for mRNA lung therapy and treating pulmonary diseases.

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Generation of Cationic Nanoliposomes for the Efficient Delivery of In Vitro Transcribed Messenger RNA

Generation of Cationic Nanoliposomes for the Efficient Delivery of In Vitro Transcribed Messenger RNA

Published on: February 1, 2019

Area of Science:

Biomaterials Science
Polymer Chemistry
Nanomedicine

Background:

Polymers are common mRNA delivery vehicles, but face challenges like poor organ selectivity and low efficacy.
Poly(amino acids), like poly(aspartic acid) (PAsp), offer biodegradability and biocompatibility for delivery applications.
The potential of aminolysis-modified PAsp for mRNA delivery requires further investigation.

Purpose of the Study:

To develop and evaluate novel poly(amino acid) derivatives for enhanced mRNA delivery.
To explore the efficacy of polyamine-aminolyzed PAsp (P-An) and its heterocyclic small molecule conjugates (P-An-M) for mRNA transfection.
To assess the in vitro and in vivo performance of these polymers for lung-targeted mRNA delivery.

Main Methods:

Synthesis of 24 polyamine-aminolyzed PAsp derivatives (P-An), including heterocyclic small molecule modifications (P-An-M).
Evaluation of in vitro mRNA transfection efficiency in 293T cells using Luc-mRNA.
Assessment of in vivo mRNA delivery, organ selectivity, and protein expression in animal models.

Main Results:

Three PAsp derivatives modified with N,N'-bis(3-aminopropyl)ethylenediamine (PDA) – P-PDA, P-PDA-I, and P-PDA-BI – demonstrated efficient Luc-mRNA transfection in 293T cells.
In vivo studies confirmed selective mRNA delivery to the lungs by P-PDA, P-PDA-I, and P-PDA-BI.
Significant protein expression was achieved in the lungs following delivery with the developed P-An-M polymers.

Conclusions:

Polyamine-aminolyzed PAsp derivatives, particularly P-PDA, P-PDA-I, and P-PDA-BI, are effective mRNA delivery platforms.
These polymers enable selective mRNA delivery to the lungs, offering a promising strategy for mRNA lung therapy.
The findings support the potential application of these novel polymers in treating pulmonary diseases.