Antisense Oligonucleotides: A Unique Treatment Approach

ABSTRACT

Synthetic Antisense oligonucleotides (ASOs) are novel and efficient laboratory tools to regulate the expression of specific genes, andhave only recently come into clinical use. These are synthetic single-stranded DNA analogs, whose sequence is complementary to atarget nucleotide and alter protein synthesis by several mechanisms. We herein provide a primer on the topic for pediatricians, as thisgroup of drugs is likely to see many more drugs for previously incurable diseases.