Nucleic acid-based therapeutics (e.g., siRNA and antisense-DNA) hold promise as the next generation of targeted therapeutic agents, most notably allowing tailored regulation of the expression of proteins within cells. Significant data show that AuraSense's Carrier-Free Gene Regulation constructs, based upon nucleic acid-functionalized gold nanoparticles, are superior to alternative platforms based on multiple key success factors, such as:
High in vivo stability. Due to their dense loading, a majority of cargo (DNA or siRNA) remains bound to the constructs inside cells, conferring nucleic acid stability and resistance to enzymatic degradation
Deliverability. For all cell types studied (e.g., neurons, tumor cell lines, etc.) the constructs demonstrate a transfection efficiency of 99% with no need for carriers or transfection agents
Therapeutic targeting. The unique target binding affinity and specificity of the constructs allow exquisite specificity for matched target sequences (i.e., limited off-target effects)
Superior efficacy. The constructs significantly outperform leading conventional transfection reagents (Lipofectamine 2000 and Cytofectin)
Low toxicity. The constructs can enter a variety of cultured cells, primary cells, and tissues with no apparent toxicity
No significant immune response.
Chemical tailorability. Any number of single or combinatorial agents (e.g., proteins, peptides, small molecules) can be used to tailor the surface of the constructs
This platform for nucleic acid-based therapeutics is applicable to numerous disease states, including inflammation and infectious disease, cancer, skin disorders and cardiovascular disease.