UCLA scientists have developed novel nanocapsules that facilitate efficient siRNA delivery into cells. The invention represents a significant advancement in realizing therapeutics based on targeted reduction of gene expression.
The novel capsulation method developed by UCLA scientists uniquely stabilizes siRNA molecules at a neutral pH, thereby preventing extracellular degradation in serum. In addition, the crosslinked polymerized shell of the nanocapsule degrades in acidic environments, like that found in late endosomes, allowing exclusive release of siRNA to occur within cells. These properties promote efficient delivery of therapeutically relevant concentrations of siRNA molecules. In practice, the proprietary nanoparticles exhibited greater knockdown of gene expression than lipofectamine in cells cultured with human serum. The results corroborate the promising utility of the nanoparticles for therapeutic siRNA delivery.
· Intracellular delivery of siRNA
o Therapeutic applications
o Research reagents
· Protects siRNA from hydrolysis
· Efficient cellular transduction of siRNA
· Preferential intracellular release of siRNA payload
The nanoparticle components and capsulation process have been optimized and tested for delivery of siRNA molecules. Proof-of-concept experiments have been performed in HEK293 cells in culture and in the presence of human serum.
|United States Of America||Issued Patent||10,568,844||02/25/2020||2010-904|
|United States Of America||Issued Patent||9,782,357||10/10/2017||2010-904|
Additional Patent Pending