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Antisense Oligonucleotides (ASOs) are short, synthetic oligonucleotides that specifically bind to target RNA sequences to modulate their expression. ASOs have applications in both research and clinical therapy, with more than eight FDA-approved products and numerous others in clinical development. The functionality of ASOs is determined by their nucleotide sequence and the chemical modifications they carry. These modifications can define the ASO’s function, and increase its stability, safety, and distribution in the body. In this chapter, we offer a comprehensive guide to designing RNAse-H1 recruiting ASOs, including guideline for sequence selection and chemical modifications. Additionally, we will outline a protocol for initial screenings of ASO libraries, providing recommendations for selection of the suitable screening platform.

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Design and Preliminary Screen of Antisense Oligonucleotides

  • 实验方案

  • 发表日期：

    2025年

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  • Nofar Mor,
  • Sharon Avkin-Nachum,
  • Dan Dominissini

摘要

Antisense Oligonucleotides (ASOs) are short, synthetic oligonucleotides that specifically bind to target RNA sequences to modulate their expression. ASOs have applications in both research and clinical therapy, with more than eight FDA-approved products and numerous others in clinical development. The functionality of ASOs is determined by their nucleotide sequence and the chemical modifications they carry. These modifications can define the ASO’s function, and increase its stability, safety, and distribution in the body. In this chapter, we offer a comprehensive guide to designing RNAse-H1 recruiting ASOs, including guideline for sequence selection and chemical modifications. Additionally, we will outline a protocol for initial screenings of ASO libraries, providing recommendations for selection of the suitable screening platform.