Peptides vs. Polymers: Searching for the Most Efficient Delivery System for Mitochondrial Gene Therapy.

ABSTRACT: Together with the nucleus, the mitochondrion has its own genome. Mutations in mitochondrial DNA are responsible for a variety of disorders, including neurodegenerative diseases and cancer. Current therapeutic approaches are not effective. In this sense, mitochondrial gene therapy emerges as a valuable and promising therapeutic tool. To accomplish this goal, the design/development of a mitochondrial-specific gene delivery system is imperative. In this work, we explored the ability of novel polymer- and peptide-based systems for mitochondrial targeting, gene delivery, and protein expression, performing a comparison between them to reveal the most adequate system for mitochondrial gene therapy. Therefore, we synthesized a novel mitochondria-targeting polymer (polyethylenimine-dequalinium) to

SUBMITTER: Faria R 

PROVIDER: S-EPMC9026848 | biostudies-literature | 2022 Mar

REPOSITORIES: biostudies-literature