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Dendrimer-formulated short activating RNAs (saRNAs) to enhance CCAAT enhancer binding protein-a (CEBPA)-dependent gene expression in liver cancer

Cell culture and rat studies suggest dendrimer-formulated saRNAs that bind target gene promoters could help treat cancer. CEPBA-saRNA was conjugated to a triethanolamine core poly(amidoamine) dendrimer for in vivo delivery. In a liver cancer cell line, CEBPA-saRNA increased secretion of albumin and decreased cell proliferation compared with a scrambled saRNA while maintaining hepatocyte function. In a cirrhotic rat model with associated liver cancer, a CEBPA-saRNA dendrimer conjugate increased levels of serum albumin and decreased tumor burden compared with saline or scrambled saRNA-dendrimer, suggesting improved liver function and antitumor activity. Next steps include developing an saRNA-nanoparticle candidate for clinical trials (see RNA is for activation, page 8).

SciBX 7(5); doi:10.1038/scibx.2014.156
Published online Feb. 6, 2014

Patents filed; available for licensing from MiNA Therapeutics Ltd.
Contact: Robert Habib, MiNA Therapeutics Ltd., London, U.K.

Reebye, V. et al. Hepatology; published online Dec. 9, 2013;
Contact: Nagy A. Habib, Imperial College London, London, U.K.

Contact: Vikash Reebye, same affiliation as above