Endosomolytic Peptides Enable the Cellular Delivery of Peptide Nucleic Acids

Abstract excerpt

<h4>ABSTRACT</h4> Precision genetic medicine enlists antisense oligonucleotides (ASOs) to bind to nucleic acid targets important for human disease. Peptide nucleic acids (PNAs) have many desirable attributes as ASOs but lack cellular permeability. Here, we use an assay based on the corrective splicing of an mRNA to assess the ability of synthetic peptides to deliver a functional PNA into a human cell. We find tha...

Topics

Open a Topic to create a Post that cites this publication.

• Disease Models, Animal
• DNA
• DNA and Nucleic Acid Chemistry
• Gene Editing
• Neurogenetic and Muscular Disorders Research
• Nucleic Acid Conformation
• Peptide Nucleic Acids
• RNA Interference and Gene Delivery

Identifiers and source

Literature Corpus work

dfccd3fd-d0b7-5ca4-a057-3cc691a0ad70

DOI

10.1101/2024.06.18.599558