Nucleic Acid Delivery

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Summary Rutgers scientists have developed a hybrid nucleic acid delivery system that combines cationic amphiphilic macromolecules (CAMs) with lipids. This system demonstrates good siRNA delivery, silencing efficiency, good stability, and low cytotoxicity, with the potential to be developed into safe and effective nucleic acid delivery vehicles for therapeutic and research purposes.   The scientists designed and synthesized novel CAMs, which are comprised of hydrophobic acyl chains and hydrophilic poly(ethylene glycol) (PEG) chains. These CAMs can self-assemble into micelles in aqueous media to present the PEG shell that increases the system’s circulation time in the blood stream. The CAMs are also biocompatible and have highly tunable structures. When used alone, the CAMs exhibit moderate gene-silencing efficiency with low cytotoxicity. Cationic lipids are the most commonly used transfection agents for gene delivery but their in-vivo utility is limited by its poor stability in the bloodstream and high cytotoxicity. By combining the novel CAMs with a mixture of cationic and fusogenic lipids (DOPE and DOTAP), the researchers were able to create a safe and stable siRNA delivery system with gene silencing efficiency comparable to Lipofectamine with improved cytocompatability.   Market Application •  RNA and DNA delivery for therapeutic utility (gene therapy, DNA vaccination, etc.) •  Research tool for nucleic acid delivery to cell   Advantages •  Biocompatibility •  Tunable chemistry •  Good nucleic acid delivery efficiency •  Good stability in blood stream