OOIR: Observatory of International Research

Papers

(The median citation count of Nucleic Acid Therapeutics is 3. 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
Design and In Vitro Evaluation of a Novel Antisense Oligonucleotide for Treatment of BK Virus Infection	20
Reversible Aptamer Staining, Sorting, and Cleaning of Cells (Clean FACS) with Antidote Oligonucleotide or Nuclease Yields Fully Responsive Cells	20
Improvements to Hybridization-Ligation ELISA Methods to Overcome Bioanalytical Challenges Posed by Novel Oligonucleotide Therapeutics	19
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
Cross-Species Translation of Biophase Half-Life and Potency of GalNAc-Conjugated siRNAs	18
Near Sequence Homology Does Not Guarantee siRNA Cross-Species Efficacy	17
Sequence- and Structure-Dependent Cytotoxicity of Phosphorothioate and 2′- O -Methyl Modified Single-Stranded Oligonucleotides	17
Targeting a Pathogenic Variant Creating an Upstream AUG in the ENG 5′ Untranslated Region with Antisense Oligonucleotides Fails to Restore Protein Expres	16
A Combined Fertility and Developmental Toxicity Study with an Antisense Oligonucleotide Targeting Murine Apolipoprotein C-III mRNA in Mice	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
RNA Interference Effectors Selectively Silence the Pathogenic Variant GNAO1 c.607 G > A In Vitro	14
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
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
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
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
Preclinical Pharmacokinetics in Tumors and Normal Tissues of the Antigene PNA Oligonucleotide MYCN-Inhibitor BGA002	9
Addressing the Challenges of Treating Patients with Heterozygous Gain of Function Mutations	9
Antisense Oligonucleotide-Based Rescue of Complex Intronic Splicing Defects in ABCA4	8
From Failure to Meet the Clinical Endpoint to U.S. Food and Drug Administration Approval: 15th Antisense Oligonucleotide Therapy Approved Qalsody (Tofersen) for Treatment of SOD	8
Development of Long Asymmetric siRNA Structure for Target Gene Silencing and Immune Stimulation in Mammalian Cells	8
Considerations for Creating the Next Generation of RNA Therapeutics: Oligonucleotide Chemistry and Innate Immune Responses to Nucleic Acids	8
Addressing the Needs of Nano-Rare Patients	8
Modulation of Gene Expression in the Eye with Antisense Oligonucleotides	8
The Quest for mRNA Vaccines	7
Experimental Model Systems Used in the Preclinical Development of Nucleic Acid Therapeutics	7
Applying Synthetic Nucleic Acid Therapeutics to Gene Activation for Haploinsufficient Diseases	6
Efficient Downregulation of Alk4 in Skeletal Muscle After Systemic Treatment with Conjugated siRNAs in a Mouse Model for Duchenne Muscular Dystrophy	6
Multiplexed In Vivo Screening Using Barcoded Aptamer Technology to Identify Oligonucleotide-Based Targeting Reagents	6
Elevation of SHANK3 Levels by Antisense Oligonucleotides Directed Against the 3′-UTR of the Human SHANK3 mRNA	6
Reduced Genotoxicity Testing Is Possible for Noncoding Oligonucleotide-Based Therapeutics Containing Well-Characterized Modifications: A European Regulatory Perspective	6
Cholesterol-Conjugated siRNA Silencing Tnf for the Treatment of Liver Macrophage-Mediated Acute Inflammation in Nonalcoholic Fatty Liver Disease	6
Aerosolized Pulmonary Delivery of mRNA Constructs Attenuates Severity of Escherichia coli Pneumonia in the Rat	6
Efficient Downregulation of Flt-1 Mediated by Splice Switching ASO in Murine Endothelial Cells	5
Impact of the Inhibition of Organic Anion Transporter on Tricyclo-DNA-Mediated Exon Skipping in the mdx Mouse Model	5
Characterizing Antibodies Targeting Antisense Oligonucleotide Phosphorothioate and 2′- O -Methoxyethyl Modifications for Intracellular Trafficking and Bi	5
Rosalind Franklin Society Proudly Announces the 2022 Award Recipient for Nucleic Acid Therapeutics	5
Nucleic Acid Therapeutics: Successes, Milestones, and Upcoming Innovation	4
mRNA Nuclear Clustering Leads to a Difference in Mutant Huntingtin mRNA and Protein Silencing by siRNAs In Vivo	4
Immunogenicity Risk Assessment for Nucleic Acid Therapeutics: A Comprehensive Evaluation for ASO, siRNA, and Nonvaccine mRNA/LNP Therapies by the IQ Consortium	4
Time Is a Critical Factor When Evaluating Oligonucleotide Therapeutics in hERG Assays	4
A Workflow for Transcriptome-Wide Assessment of Antisense Oligonucleotide Selectivity	4
It is Time to Revisit miRNA Therapeutics	4
Antisense Oligonucleotide Quantification via Splint-Ligation PCR Assay in Nonhuman Primate Central Nervous System Tissues and Biofluids	4
Report of the European Medicines Agency Conference on RNA-Based Medicines	4
Developmental and Reproductive Toxicity Testing Strategies for Oligonucleotides: A Workshop Proceedings	3
Understanding and Rescuing the Splicing Defect Caused by the Frequent ABCA4 Variant c.4253+43G>A Underlying Stargardt Disease	3
Antisense Oligonucleotide Rescue of Deep-Intronic Variants Activating Pseudoexons in the 6-Pyruvoyl-Tetrahydropterin Synthase Gene	3
Acknowledgment of Reviewers 2022	3
Antisense Oligonucleotide STK-002 Increases OPA1 in Retina and Improves Mitochondrial Function in Autosomal Dominant Optic Atrophy Cells	3
Nucleic Acid Aptamers Increase the Anticancer Efficiency and Reduce the Toxicity of Cisplatin-Arabinogalactan Conjugates In Vivo	3
Systematic Analysis of Chemical Modifications of Phosphorothioate Antisense Oligonucleotides that Modulate Their Innate Immune Response	3
Considerations in the Preclinical Assessment of the Safety of Antisense Oligonucleotides	3
“Goldilocks Modifications” for mRNA Therapeutics Won the Nobel Prize	3

Positive Allosteric Modulation of Antithrombin's Inhibitory Activity by RNA Aptamers	3
Host-Directed Targeting of LincRNA-MIR99AHG Suppresses Intracellular Growth of Mycobacterium tuberculosis	3
Inflammatory Non-CpG Antisense Oligonucleotides Are Signaling Through TLR9 in Human Burkitt Lymphoma B Bjab Cells	3
Chemical Modifications and Design Influence the Potency of Huntingtin Anti-Gene Oligonucleotides	3