Enhanced Accumulation of Heteroduplex Oligonucleotides in Dystrophin-Deficient Skeletal Muscles by Single Oligonucleotide-Loaded Unit Polyion Complexes.

ABSTRACT: Efficient delivery of oligonucleotide drugs to muscle tissues remains a significant challenge in nanomedicine and oligonucleotide therapeutics. A primary obstacle is the blood-muscle barrier, a continuous endothelium within muscle tissues that impedes the extravasation of conventional nanomedicines, typically ranging from a few tens of nanometers to 100 nm. To address this challenge, we developed an ultrasmall oligonucleotide nanomedicine, termed the unit polyion complex (uPIC), using a single molecular oligonucleotide with Y-shaped block catiomers. Heteroduplex oligonucleotide (HDO)-loaded uPICs with hydrodynamic diameters of approximately 20 nm were prepared. Comparative analysis revealed that HDO-loaded uPICs accumulated significantly more in the dystrophin-deficient quadriceps of Duche

SUBMITTER: Chaya H 

PROVIDER: S-EPMC12355270 | biostudies-literature | 2025 Aug

REPOSITORIES: biostudies-literature