Reagents for improved DNA lipofection, depot anti-inflammation, and/or topical antimicrobial therapy

Researchers in the Diamond Lab have developed a versatile new class of pharmacologically-active molecules with multiple applications. These molecules deliver plasmids (pDNA), adenovirus (AdV), and adeno-associated virus (AAV) to cultured cells and to pulmonary epithelium in vivo.  In addition to their gene delivery capabilities, the structure of the molecules allows them to function as slow-release prodrugs for local treatment of vector-associated inflammation. Importantly, this therapeutic effect may synergize with the gene that is delivered. 

The anti-inflammatory effect of the cationic steroids does not require DNA, and as the prodrugs degrade, they break down into non-toxic materials with pharmacological activity. The molecules can be readily synthesized in one-step from inexpensive precursors in a scalable reaction and purified in gram quantities. Additionally, the cationic steroids have potent antimicrobial activity against gram-positive and gram-negative bacteria, including clinical strains of MRSA and pseudomonas.

• Efficient delivery of genes to cultured cells
• Synergistic combination of gene delivery and anti-inflammatory effects to enhance gene therapy
• Rapid production of compound in large quantities
• Potent and rapid bactericidal activity against drug-resistant bacteria

• Gene therapy via non-viral gene delivery
• Slow-release therapeutics for treatment of inflammation
• Enhancer of DNA vaccines
• Pulmonary infections