Lipophilic Peptide Dendrimers for Delivery of Splice-Switching Oligonucleotides.

ABSTRACT: Non-viral transfection reagents are continuously being developed in attempt to replace viral vectors. Among those non-viral vectors, dendrimers have gained increasing interest due to their unique molecular structure and multivalency. However, more improvements are still needed to achieve higher efficacy and lower toxicity. In this study, we have examined 18 peptide dendrimers conjugated to lipophilic moieties, such as fatty acids or hydrophobic amino acids, that were previously explored for siRNA. Reporter cells were employed to investigate the transfection of single strand splice-switching oligonucleotides (ONs) using these peptide dendrimers. Luciferase level changes reflecting efficiency varied with amino acid composition, stereochemistry, and complexation media used. 3rd generation pep

SUBMITTER: Daralnakhla H 

PROVIDER: S-EPMC7831936 | biostudies-literature | 2021 Jan

REPOSITORIES: biostudies-literature