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Antisense Oligonucleotides: A Unique Treatment Approach

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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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Authors and Affiliations

  1. Department of Pediatrics, Maulana Azad Medical College (University of Delhi), Delhi, 110002, India

    Abhinaya V. Krishnan & Devendra Mishra

Authors
  1. Abhinaya V. Krishnan
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  2. Devendra Mishra
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Contributions

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.

Corresponding author

Correspondence to Devendra Mishra.

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Competing interest

None stated.

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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

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