Evidence mapPaperPMID 39653607Full record

ReviewExpert opinion on drug discovery2025

Evolution of antisense oligonucleotides: navigating nucleic acid chemistry and delivery challenges.

Ruchi Ruchi, Govind Mukesh Raman, Vikas Kumar, Raman Bahal

Abstract readReview
In one paragraph

Review in Expert opinion on drug discovery, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers.

0numbers the graph read from it
0cells of the map it votes in
8citing papers in PubMed
field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

8 citing papers in PubMed.

  1. Review
  2. Article
  3. Advances in antisense oligonucleotide treatment for cancer.Japanese journal of clinical oncology · 2026
    Review
  4. Review
  5. Article
  6. Article
  7. Review
  8. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

4 authors.

Ruchi RuchiDepartment of Pharmaceutical Sciences, University of Connecticut, Storrs, CT, USA.
Govind Mukesh RamanDepartment of Pharmaceutical Sciences, University of Connecticut, Storrs, CT, USA.
Vikas KumarDepartment of Pharmaceutical Sciences, University of Connecticut, Storrs, CT, USA.ORCID 0000-0002-8004-4155
Raman BahalDepartment of Pharmaceutical Sciences, University of Connecticut, Storrs, CT, USA.ORCID 0000-0002-4514-8004

Funding

Developing next generation synthetic nucleic acid analogous for sickle cell disease gene editingR01HL147028 · NHLBI · UNIVERSITY OF CONNECTICUT STORRS · PI Raman Bahal · 2022 to 2024
$1.2M
Targeting microRNAs in the tumor microenvironment with pHLIP conjugated next generation chemically modified PNAsR01CA241194 · NCI · BETH ISRAEL DEACONESS MEDICAL CENTER · PI FRANK J. SLACK · 2022 to 2024
$1.1M
NCI NIH HHS R01 CA241194NHLBI NIH HHS R01 HL147028
6 · The paper itself

Abstract

introductionAntisense oligonucleotide (ASO) was established as a viable therapeutic option for genetic disorders. ASOs can target RNAs implicated in various diseases, including upregulated mRNA and pre-mRNA undergoing abnormal alternative splicing events. Therapeutic applications of ASOs have been proven with the Food and Drug Administration approval of several drugs in recent years. Earlier enzymatic stability and delivery remains a big challenge for ASOs. Introducing new chemical modifications and new formulations resolving the issues related to the nuclease stability and delivery of the ASOs. Excitingly, ASOs-based bioconjugates that target the hepatocyte have gained much attraction. Efforts are ongoing to increase the therapeutic application of the ASOs to the extrahepatic tissue as well. AREA COVERED: We have briefly discussed the mechanism of ASOs, the development of new chemistries, and delivery strategies for ASO-based drug discovery and development. The discussion focuses more on the already approved ASOs and those in the clinical development stage. EXPERT OPINION: To expand the clinical application of ASOs, continuous effort is required to develop precise delivery strategies for targeting extrahepatic tissue to minimize the off-target effects.

Indexed as

Drug Delivery SystemsDrug DevelopmentOligonucleotides, AntisenseAnimalsDrug DiscoveryDrug StabilityGenetic Diseases, InbornHumansNucleic AcidsNucleic AcidsOligonucleotides, AntisenseAntisense oligonucleotidesbioconjugatesmRNAnucleic acid chemistrytargeted delivery

Identifiers

PMID39653607
PMCPMC11823135

What Socratic holds

Textmetadata
LicenceTDM
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.