Lipid-based transfection and electroporation are widely utilized, conventional methods to deliver siRNA into the cells. However, in many primary cells, particularly immune cells, hematopoietic cells and neurons, lipid reagents and electroporation are associated with high toxicity and poor transfection efficiency. Alternative delivery methods, such as viral vectors, require laborious optimization and viral production steps, and carry associated risk of genome integration.
AUMsilence Antisense Oligonucleotides are modified with 3rd generation chemical modifications that enhances the intracellular stability of the oligos, as well as their binding to the target mRNA. The AUMsilence ASOs can be self-delivered into cells without any transfection reagents, as well as in animals, without the need of special delivery formulations.