Development of Peptide-Based Nanoparticles for Mitochondrial Plasmid DNA Delivery

A mitochondrion is a cellular organelle able to produce energy in the form of adenosine triphosphate (ATP). As nucleus, mitochondria contain their own genome: mitochondrial DNA (mtDNA). This genome particularly susceptible mutations that are at basis multitude disorders, especially those affecting heart, central nervous system and muscles. Conventional clinical practice applied diseases very limited ineffective; clear need for innovative therapies demonstrated. Gene therapy seems be promising approach. The use as therapeutic, optimized by peptide-based complexes with targeting, can seen powerful tool reestablishment normal function. In line this requirement, work first time, mitochondrial-targeting sequence (MTS) has been incorporated into previously researched peptides, confer on them targeting ability. These peptides were then considered complex plasmid (pDNA) which contains gene ND1 (mitochondrially encoded NADH dehydrogenase 1 protein), aiming formation nanoparticles. Currently, one most advanced bioengineered vectors conducting research expression. formed characterized terms pDNA complexation capacity, morphology, size, surface charge cytotoxic profile. data revealed developed carriers possess suitable properties delivery. Furthermore, vitro studies illustrated ability novel peptide/pDNA complexes. comparison between different ones target mitochondria. may contribute optimization non-viral systems mitochondria, instigating progress therapy.