By a News Reporter-Staff News Editor at Life Science Weekly -- Investigators publish new report on DNA Research. According to news reporting out of Stanford, California, by NewsRx editors, research stated, "Gene therapy provides a powerful tool for regulating cellular processes and tissue repair. Minicircle (MC) DNA are supercoiled DNA molecules free of bacterial plasmid backbone elements and have been reported to enhance prolonged gene expression compared to conventional plasmids."
Our news journalists obtained a quote from the research from Stanford University, "Despite the great promise of MC DNA for gene therapy, methods for safe and efficient MC DNA delivery remain lacking. To overcome this bottleneck, here we report the development of a poly(beta-amino ester) (PBAE)-based, biodegradable nanoparticulate platform for efficient delivery of MC DNA driven by a Ubc promoter in vitro and in vivo. By synthesizing and screening a small library of 18 PBAE polymers with different backbone and end-group chemistry, we identified lead cationic PBAE structures that can complex with minicircle DNA to form nanoparticles, and delivery efficiency can be further modulated by tuning PBAE chemistry. Using human embryonic kidney 293 cells and mouse embryonic fibroblasts as model cell types, we identified a few PBAE polymers that allow efficient MC delivery at levels that are comparable or even surpassing Lipofectamine 2000. The biodegradable nature of PBAE-based nanoparticles facilitates in vivo applications and clinical translation. When injected via intraperitoneal route in vivo, MC alone resulted in high transgene expression, and a lead PBAE/MC nanoparticle formulation achieved a further 2-fold increase in protein expression compared to MC alone."
According to the news editors, the research concluded: "Together, our results highlight the promise of PBAE-based nanoparticles as promising nonviral gene carriers for MC delivery, which may provide a valuable tool for broad applications of MC DNA-based gene therapy."
For more information on this research see: Development of Poly(beta-amino ester)-Based Biodegradable Nanoparticles for Nonviral Delivery of Minicircle DNA. ACS Nano, 2013;7(8):7241-7250. ACS Nano can be contacted at: Amer Chemical Soc, 1155 16TH St, NW, Washington, DC 20036, USA. (American Chemical Society - www.acs.org; ACS Nano - www.pubs.acs.org/journal/ancac3)
Our news journalists report that additional information may be obtained by contacting M. Keeney, Stanford University, Dept. of Bioengn, Stanford, CA 94305, United States. Additional authors for this research include S.G. Ong, A. Padilla, Z.Y. Yao, S. Goodman, J.C. Wu and F. Yang (see also DNA Research).
Keywords for this news article include: Therapy, Stanford, Chemicals, Chemistry, California, DNA Research, Nanoparticle, United States, Nanotechnology, Emerging Technologies, North and Central America
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