Brain Gene Silencing with Cationic Amino-Capped Poly(ethylene glycol) Polyplexes.

ABSTRACT: Therapeutic gene silencing in the brain is usually achieved using highly invasive intracranial administration methods and/or comparatively toxic vectors. In this work, we use a relatively biocompatible vector: poly(ethylene glycol) star-shaped polymer capped with amine groups (4APPA) via the nose to brain route. 4APPA complexes anti- itchy E3 ubiquitin protein ligase (anti-ITCH) siRNA to form positively charged (zeta potential +15 ± 5 mV) 150 nm nanoparticles. The siRNA-4APPA polyplexes demonstrated low cellular toxicity (IC50 = 13.92 ± 6 mg mL^-1) in the A431 cell line and were three orders of magnitude less toxic than Lipofectamine 2000 (IC50 = 0.033 ± 0.04 mg mL^-1) in this cell line. Cell association and uptake of fluorescently labelled siRNA bound to siRNA-4APPA na

SUBMITTER: Alamoudi AA 

PROVIDER: S-EPMC9496157 | biostudies-literature | 2022 Sep

REPOSITORIES: biostudies-literature