Multimeric Conjugates Using Engineered Peptide Scaffolds for Efficient siRNA Delivery.

ABSTRACT: Oligonucleotide therapeutics (ONT) traditionally involve a single targeting moiety per oligonucleotide when conjugated for organ delivery. Multimerization represents a novel approach by connecting multiple ONTs to a single scaffold, thereby influencing the drug's activity and biophysical properties in vivo. Recently, others have demonstrated the efficacy of this strategy, showing enhanced tissue retention and extended silencing with the capability to target multiple genes simultaneously. The investigation of diverse multimeric designs is thus an exciting opportunity to explore the delivery of the ONT. In this study, we engineered a versatile peptide branching unit able to link up to four small interfering RNAs together. We conjugated a GalNAc targeting moiety to these scaffolds for

SUBMITTER: Vicentini Q 

PROVIDER: S-EPMC12183678 | biostudies-literature | 2025 Jun

REPOSITORIES: biostudies-literature