Multifunctional Polypeptide-Based Nanoconjugates for Targeted Mitochondrial Delivery and Nonviral Gene Therapy.

ABSTRACT: Despite recent advances in nanomedicine, developing multifunctional nanocarriers capable of targeted subcellular delivery and efficient gene therapy remains a significant challenge. This study reports the design, synthesis, and evaluation of a novel multifunctional polypeptide-based nanoconjugate that addresses this gap using sequential delivery, combining mitochondrial targeting and nonviral gene therapy. We engineered a poly-l-ornithine-based, polyethylene glycol-modified carrier and introduced a novel custom-designed trivalent compound (TRV3) into the structure. TRV3, conjugated to the polypeptide carrier via a redox-sensitive disulfide linker, incorporates the well-described triphenylphosphonium moiety (TPP) for mitochondrial targeting and a Cy5 fluorophore as a model drug. The resulti

SUBMITTER: Pegoraro C 

PROVIDER: S-EPMC11866743 | biostudies-literature | 2025 Feb

REPOSITORIES: biostudies-literature