Abstract

Retinoblastoma (RB) is the main pediatric intraocular tumor in children. It is caused by the mutation of tumor suppressor gene RB transcriptional corepressor 1 (RB1). Herein, poly(ethylene glycol) grafted branched polyethyleneimine (hyperbranched-star PEI-g-PEG) was taken as the polycationic gene carrier for preparing the nonviral vectors with efficient uptake and hypotoxicity for potential retinoblastoma gene therapy application. The PEI-g-PEG can condense the genes effectively and the cationic, nano-scaled complexes with PEG shells were obtained by optimization the composition proportions. The cell behaviors investigations indicated that the PEGylation of PEI can greatly decrease the cytotoxicity, and such gene vector has efficient endocytosis effects. The gene transfection results indicated that different functional proteins can be expressed in the retinoblastoma cells when using the functional genes were encoded in the PEI-g-PEG based nonviral gene vectors. These results imply that the hyperbranched-star PEG-g-PEI is a promising nonviral carrier for gene delivery in retinoblastoma.

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