Abstract
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.
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Both authors were involved in planning the manuscript and review of literature. Initial draft: AVK; Final manuscript: AVK, DM. Both authors approved the final manuscript.
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Krishnan, A.V., Mishra, D. Antisense Oligonucleotides: A Unique Treatment Approach. Indian Pediatr 57, 165–171 (2020). https://doi.org/10.1007/s13312-020-1736-7
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DOI: https://doi.org/10.1007/s13312-020-1736-7