Next-generation antisense oligonucleotide delivery approaches

摘要

Antisense oligonucleotides (ASOs) have enormous potential for the treatment of genetic disorders. Targeted drug delivery to disease-relevant tissues remains the greatest obstacle for the development of ASO therapeutics for a broader spectrum of diseases. Next-generation delivery approaches like spherical nucleic acids (SNAs) and ASO conjugates aim to combat ASO delivery limitations, and thereby decrease the effective dose, dose frequency, and off-target effects of ASOs. SNAs and ASO conjugated drugs have progressed to clinical trials for diseases of the skin and liver, organs that were historically challenging to target with ASOs. In this article, we will discuss how innovative approaches like SNAs and conjugates may improve drug delivery and expand the therapeutic potential of ASOs for many more diseases.