PEG-PEI from PolySciTech used in development of Blood-Brain-Barrier crossing nanoparticles for gene therapy

 

Many central-nervous related diseases (Parkinson’s, Alzheimer’s, ALS, etc.) are difficult to treat due in part due to the design of the body which prevents many molecules from crossing over into the brain tissue from the blood-stream. Although the Blood-Brain-Barrier (BBB) is a necessary component to human survival as it protects the brain from potentially damaging chemicals it makes treating CNS diseases difficult. Recently, researchers at Tokyo University and Teikyo University (Japan) used PEG-PEI (AK086) from PolySciTech (www.polyscitech.com) to create gene-loaded nanoparticles for crossing the BBB. This research holds promise to improve therapy options against a range of neural diseases in the future. Read more: Endo-Takahashi, Yoko, Ryo Kurokawa, Kanako Sato, Nao Takizawa, Fumihiko Katagiri, Nobuhito Hamano, Ryo Suzuki et al. "Ternary Complexes of pDNA, Neuron-Binding Peptide, and PEGylated Polyethyleneimine for Brain Delivery with Nano-Bubbles and Ultrasound." Pharmaceutics 13, no. 7 (2021): 1003. https://www.mdpi.com/1999-4923/13/7/1003

“Abstract: In brain-targeted delivery, the transport of drugs or genes across the blood−brain barrier (BBB) is a major obstacle. Recent reports found that focused ultrasound (FUS) with microbubbles enables transient BBB opening and improvement of drug or gene delivery. We previously developed nano-sized bubbles (NBs), which were prepared based on polyethylene glycol (PEG)-modified liposomes containing echo-contrast gas, and showed that our NBs with FUS could also induce BBB opening. The aim of this study was to enhance the efficiency of delivery of pDNA into neuronal cells following transportation across the BBB using neuron-binding peptides. This study used the RVG-R9 peptide, which is a chimeric peptide synthesized by peptides derived from rabies virus glycoprotein and nonamer arginine residues. The RVG peptide is known to interact specifically with the nicotinic acetylcholine receptor in neuronal cells. To enhance the stability of the RVG-R9/pDNA complex in vivo, PEGylated polyethyleneimine (PEG-PEI) was also used. The ternary complexes composed of RVG-R9, PEG-PEI, and pDNA could interact with mouse neuroblastoma cells and deliver pDNA into the cells. Furthermore, for the in vivo experiments using NBs and FUS, gene expression was observed in the FUS-exposed brain hemispheres. These results suggest that this systemic gene delivery system could be useful for gene delivery across the BBB. Keywords: nanobubble; ultrasound; brain; gene delivery”