OOIR: Observatory of International Research

Papers

(The TQCC of Nucleic Acid Therapeutics is 10. The table below lists those papers that are above that threshold based on CrossRef citation counts [max. 250 papers]. The publications cover those that have been published in the past four years, i.e., from 2022-05-01 to 2026-05-01.)

Article	Citations
Assessing Hybridization-Dependent Off-Target Risk for Therapeutic Oligonucleotides: Updated Industry Recommendations	129
Knockdown of Circular RNAs Using LNA-Modified Antisense Oligonucleotides	103
Combined 20-Hydroxyecdysone and Antisense-Mediated Exon Skipping Improve Functional Outcomes in a Mouse Model of Duchenne Muscular Dystrophy	87
SIDT2 Inhibits Phosphorothioate Antisense Oligonucleotide Activity by Regulating Cellular Localization of Lysosomes	77
Quantification of mRNA Decay Rates in HeLa and SH-SY5Y Cell Lines Reveals Novel Properties of Membrane Protein Coding Transcripts	48
Mechanistic Insights into Hybridization-Based Off-Target Activity of GalNAc-siRNA Conjugates	47
Therapeutic siRNA Loaded to RISC as Single and Double Strands Requires an Appropriate Quantitative Assay for RISC PK Assessment	42
Correction to: Understanding and Rescuing the Splicing Defect Caused by the Frequent ABCA4 Variant c.4253 + 43G>A Underlyin	38
Challenges of Assessing Exon 53 Skipping of the Human DMD Transcript with Locked Nucleic Acid-Modified Antisense Oligonucleotides in a Mouse Model for Du	30
Screening Splice-Switching Antisense Oligonucleotides in Pancreas-Cancer Organoids	24
Reversible Aptamer Staining, Sorting, and Cleaning of Cells (Clean FACS) with Antidote Oligonucleotide or Nuclease Yields Fully Responsive Cells	20
Design and In Vitro Evaluation of a Novel Antisense Oligonucleotide for Treatment of BK Virus Infection	20
Inhibition of Epidermal Growth Factor Receptor Signaling by Antisense Oligonucleotides as a Novel Approach to Epidermal Growth Factor Receptor Inhibition	19
Considerations for the Terminal Sterilization of Oligonucleotide Drug Products	19
Improvements to Hybridization-Ligation ELISA Methods to Overcome Bioanalytical Challenges Posed by Novel Oligonucleotide Therapeutics	19
Cross-Species Translation of Biophase Half-Life and Potency of GalNAc-Conjugated siRNAs	18
Sequence- and Structure-Dependent Cytotoxicity of Phosphorothioate and 2′- O -Methyl Modified Single-Stranded Oligonucleotides	17
Near Sequence Homology Does Not Guarantee siRNA Cross-Species Efficacy	17
A Combined Fertility and Developmental Toxicity Study with an Antisense Oligonucleotide Targeting Murine Apolipoprotein C-III mRNA in Mice	16
Targeting a Pathogenic Variant Creating an Upstream AUG in the ENG 5′ Untranslated Region with Antisense Oligonucleotides Fails to Restore Protein Expres	16
Aptamers Targeting Hallmark Proteins of Neurodegeneration	15
RIG-I-Mediated Innate Immune Stimulation by Chemically Synthesized Long Double-Stranded RNAs Is Structure and Sequence Dependent	15
Sequence-Controlled Spherical Nucleic Acids: Gene Silencing, Encapsulation, and Cellular Uptake	14
DNAzymes: Expanding the Potential of Nucleic Acid Therapeutics	14
Rapid and Reliable Quantification of Prime Editing Targeting Within the Porcine ABCA4 Gene Using a BRET-Based Sensor	14

Peptide Nucleic Acid-Mediated Regulation of CRISPR-Cas9 Specificity	14
Programmable RNA Editing via Adenosine Deaminase Acting on RNA Enzymes: Current Advances and Clinical Potential	14
RNA Interference Effectors Selectively Silence the Pathogenic Variant GNAO1 c.607 G > A In Vitro	14
Levels of Exon-Skipping Are Not Artificially Overestimated Because of the Increased Affinity of Tricyclo-DNA-Modified Antisense Oligonucleotides to the Target DMD	13
Binary Antisense Oligonucleotide Agent for Cancer Marker-Dependent Degradation of Targeted RNA	12
Biodistribution of Radioactively Labeled Splice Modulating Antisense Oligonucleotides After Intracerebroventricular and Intrathecal Injection in Mice	11
Hepatotoxicity Reduction Profiles of Antisense Oligonucleotides Containing Amido-Bridged Nucleic Acid and 2′-O,4′-C-Spirocyclopropylene Bridged Nucleic Acid	11
New Oligonucleotide 2′-O-Alkyl N3′→P5′ (Thio)-Phosphoramidates as Potent Antisense Agents: Physicochemical Properties and Biological Activity	11
Binding and Ligand Activation Driven Enrichment–Directed Evolution of SaCas9 gRNAs Improves Gene Editing Efficiency	10
Systematic Evaluation of Position-Specific Tolerability of Seven Backbone and Ribose Modifications in Fully Chemically Stabilized siRNAs	10
Splice-Switching Antisense Oligonucleotides Correct Phenylalanine Hydroxylase Exon 11 Skipping Defects and Rescue Enzyme Activity in Phenylketonuria	10
Opportunities for More Tailored Approaches to Genotoxicity Testing and Carcinogenicity Strategy for Oligonucleotide Therapeutics: Outcome of an Industry Survey	10