259. Mitochondria-Targeted Delivery of siRNA by Amphiphilic Nanovectors Self-Assembled from a Cationic [4]Helicene-Squalene Ester
     Lamiaa M. A. Ali, Dan–Dan Su, Rebecca Mucci, Kévin Martin, Mihail Barboiu, Jérôme Lacour, Nadir Bettache, Sébastien Ulrich
     ChemBioChem 2025, 26, e202500537

The active delivery of therapeutic nucleic acids with subcellular precision is an open challenge. Herein, a small conjugate molecule, combining a cationic [4]helicene moiety with a lipophilic squalene tail, yields amphiphilic nanoassemblies capable of effectively complexing siRNA within the mitochondria. A proof-of-concept in HCT 116 colorectal cancer cells was evidenced by Western blot for the delivery of siRNA silencing the protein expression of the mitochondria-encoded cytochrome c oxidase subunit 1 gene.

DOI : 10.1002/cbic.202500537