JoLynn B. Giancola

Nov 11, 2024 | pubs.rsc.org | JoLynn B. Giancola |Ronald T. Raines

Endosomolytic Peptides Enable the Cellular Delivery of Peptide Nucleic Acids 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.

Jun 18, 2024 | biorxiv.org | JoLynn B. Giancola |Ronald T. Raines

AbstractPrecision 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 that the endosomolytic peptides L17E and L17ER4 are highly efficacious delivery vehicles.