Noninvasive delivery of oligonucleotide by penetratin-modified polyplexes to inhibit protein expression of intraocular tumor

We have developed an antisense oligonucleotide delivery polyplex (PG5/ASO/Pene) by using PAMAM G5 as the cationic carrier and penetratin as the permeation enhancer. The self-assembled polyplex was stabilized via non-covalent bonding. In vitro assays revealed that PG5/ASO/Pene polyplex exhibited efficient down-regulation of luciferase expression, which was further demonstrated in subcutaneous tumor model. Notably, in orthotopic tumor model, PG5/ASO/Pene polyplex inhibited protein expression of intraocular tumor via topical instillation.341