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.
FANA ASOs are uniquely designed and manufactured using 2′-deoxy-2′- fluoro-β-d-arabinonucleic acid (FANA) chemical modifications that enhance intracellular stability of the oligos, providing very high specificity as well as high binding affinity to the target mRNA. The FANA modifications also allow for the oligos to be self-delivered into cells without any transfection reagents or electroporation. FANA ASOs can be used for animal studies without the need of delivery formulations or conjugates.