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Updated: Jun 21, 2026

Sequence-specific and Selective Recognition of Double-stranded RNAs over Single-stranded RNAs by Chemically Modified Peptide Nucleic Acids

Sequence-specific and Selective Recognition of Double-stranded RNAs over Single-stranded RNAs by Chemically Modified Peptide Nucleic Acids

Published on: September 21, 2017

Antisense oligonucleotides - the way forward.

C Wahlestedt¹, L Good

¹Center for Genomics Research, Karolinska Institutet, S-171 77 Stockholm, Sweden. claes.wahlestedt@cgr.ki.se

Current Opinion in Drug Discovery & Development

|August 4, 2009

Summary

This summary is machine-generated.

Antisense oligonucleotides offer promising therapeutic potential, but challenges remain in achieving high in vivo efficacy without toxicity. Further research into novel analogs, improved delivery, and standardized assays is crucial for advancing this drug class.

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Intrathecal Delivery of Antisense Oligonucleotides in the Rat Central Nervous System

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Published on: October 29, 2019

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Last Updated: Jun 21, 2026

Sequence-specific and Selective Recognition of Double-stranded RNAs over Single-stranded RNAs by Chemically Modified Peptide Nucleic Acids

Sequence-specific and Selective Recognition of Double-stranded RNAs over Single-stranded RNAs by Chemically Modified Peptide Nucleic Acids

Published on: September 21, 2017

Intrathecal Delivery of Antisense Oligonucleotides in the Rat Central Nervous System

Intrathecal Delivery of Antisense Oligonucleotides in the Rat Central Nervous System

Published on: October 29, 2019

Area of Science:

Biotechnology
Drug Discovery
Molecular Biology

Background:

Antisense oligonucleotides (ASOs) present a promising avenue for novel drug development, leveraging simple base-pairing rules.
The field has seen functional genomics applications and recent clinical successes, highlighting ASO therapeutic potential.
Despite progress, significant challenges persist regarding design uncertainties and unforeseen biological effects.

Purpose of the Study:

To address current design uncertainties and emergent unforeseen effects associated with antisense oligonucleotides.
To identify novel oligonucleotide analogs that achieve high in vivo efficacy with minimal toxicity.
To improve target site selection, delivery, and bioavailability of antisense agents.

Main Methods:

Review and analysis of existing antisense oligonucleotide design principles and clinical outcomes.
Exploration of advancements in oligonucleotide analog development for enhanced therapeutic properties.
Emphasis on the need for improved in vitro and in vivo assays and standardized laboratory controls.

Main Results:

Novel oligonucleotide analogs with potential for high in vivo efficacy and reduced toxicity are emerging.
Advances in understanding and mitigating design uncertainties and unforeseen effects are being made.
Progress in improving delivery and bioavailability properties is evident.

Conclusions:

Standardized assays and controls are essential for reproducible and comparable results across laboratories.
Further development of antisense agents requires expertise in target selection and delivery optimization.
Antisense oligonucleotides hold significant promise for broad research and therapeutic applications with continued innovation.