Antisense Oligonucleotides: A Unique Treatment Approach.

Abhinaya V Krishnan, Devendra Mishra

Indian pediatrics 2020 Feb 15

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