PBAE 457

PBAE 457 is a specific poly(β-amino ester) (PBAE) nanoparticle formulation developed for non-viral gene therapy. It was identified through a high-throughput screen of biodegradable cationic polymers for its high transfection efficiency and low toxicity. In preclinical studies, PBAE 457 has been used to deliver wild-type p53 (WT p53) genetic cargo to small cell lung cancer (SCLC) cells, aiming to restore tumor suppressor function. The nanoparticles encapsulate DNA to form biodegradable cationic complexes that facilitate cellular uptake and endosomal release. Research conducted at Johns Hopkins University demonstrated that PBAE 457-mediated delivery of p53-GFP plasmids induced cell cycle arrest and anti-proliferative effects in p53-mutant SCLC models both in vitro and in vivo.

Other names

poly(beta-amino ester) 457