OOIR: Observatory of International Research

Papers

(The median citation count of Nucleic Acid Therapeutics is 4. 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 2022-01-01 to 2026-01-01.)

Article	Citations
Combined 20-Hydroxyecdysone and Antisense-Mediated Exon Skipping Improve Functional Outcomes in a Mouse Model of Duchenne Muscular Dystrophy	106
Assessing Hybridization-Dependent Off-Target Risk for Therapeutic Oligonucleotides: Updated Industry Recommendations	101
SIDT2 Inhibits Phosphorothioate Antisense Oligonucleotide Activity by Regulating Cellular Localization of Lysosomes	79
Knockdown of Circular RNAs Using LNA-Modified Antisense Oligonucleotides	78
Combining Heparin and a FX/Xa Aptamer to Reduce Thrombin Generation in Cardiopulmonary Bypass and COVID-19	76
Correction to: Understanding and Rescuing the Splicing Defect Caused by the Frequent ABCA4 Variant c.4253 + 43G>A Underlying Stargardt Disease, by Nuria Suárez-Herrera et al., Nucleic	63
Challenges of Assessing Exon 53 Skipping of the Human DMD Transcript with Locked Nucleic Acid-Modified Antisense Oligonucleotides in a Mouse Model for Duchenne Muscular Dystrophy	62
Quantification of mRNA Decay Rates in HeLa and SH-SY5Y Cell Lines Reveals Novel Properties of Membrane Protein Coding Transcripts	49
Therapeutic siRNA Loaded to RISC as Single and Double Strands Requires an Appropriate Quantitative Assay for RISC PK Assessment	46
Mechanistic Insights into Hybridization-Based Off-Target Activity of GalNAc-siRNA Conjugates	41
Screening Splice-Switching Antisense Oligonucleotides in Pancreas-Cancer Organoids	40
Inhibition of Epidermal Growth Factor Receptor Signaling by Antisense Oligonucleotides as a Novel Approach to Epidermal Growth Factor Receptor Inhibition	32
Reversible Aptamer Staining, Sorting, and Cleaning of Cells (Clean FACS) with Antidote Oligonucleotide or Nuclease Yields Fully Responsive Cells	30
Design and In Vitro Evaluation of a Novel Antisense Oligonucleotide for Treatment of BK Virus Infection	29
Optimization of RNA Aptamer SELEX Methods: Improved Aptamer Transcript 3′-End Homogeneity, PAGE Purification Yield, and Target-Bound Aptamer RNA Recovery	29
17th Annual Meeting of the Oligonucleotide Therapeutics Society: A Tribute to Bob Letsinger, Progress in N = 1 Treatments, and Successful First In-Humans CRISPR Trials	27
Improvements to Hybridization-Ligation ELISA Methods to Overcome Bioanalytical Challenges Posed by Novel Oligonucleotide Therapeutics	24
Considerations for the Terminal Sterilization of Oligonucleotide Drug Products	21
Cross-Species Translation of Biophase Half-Life and Potency of GalNAc-Conjugated siRNAs	19
Sequence- and Structure-Dependent Cytotoxicity of Phosphorothioate and 2′-O-Methyl Modified Single-Stranded Oligonucleotides	19
Near Sequence Homology Does Not Guarantee siRNA Cross-Species Efficacy	16
Sequence-Controlled Spherical Nucleic Acids: Gene Silencing, Encapsulation, and Cellular Uptake	16
Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A First-in-human, Multicenter, Two-Part, Open-Label, Phase 1/2 Tr	16
A Combined Fertility and Developmental Toxicity Study with an Antisense Oligonucleotide Targeting Murine Apolipoprotein C-III mRNA in Mice	16
Individualized Antisense Oligonucleotide Therapies: How to Approach the Challenge of Manufacturing These Oligos from a Chemistry, Manufacturing, and Control-Regulatory Standpoint	15

RIG-I-Mediated Innate Immune Stimulation by Chemically Synthesized Long Double-Stranded RNAs Is Structure and Sequence Dependent	15
Targeting a Pathogenic Variant Creating an Upstream AUG in the ENG 5′ Untranslated Region with Antisense Oligonucleotides Fails to Restore Protein Expres	15
DNAzymes: Expanding the Potential of Nucleic Acid Therapeutics	14
Binary Antisense Oligonucleotide Agent for Cancer Marker-Dependent Degradation of Targeted RNA	14
Aptamers Targeting Hallmark Proteins of Neurodegeneration	14
RNA Interference Effectors Selectively Silence the Pathogenic Variant GNAO1 c.607 G > A In Vitro	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	13
Levels of Exon-Skipping Are Not Artificially Overestimated Because of the Increased Affinity of Tricyclo-DNA-Modified Antisense Oligonucleotides to the Target DMD Exon	13
New Oligonucleotide 2′-O-Alkyl N3′→P5′ (Thio)-Phosphoramidates as Potent Antisense Agents: Physicochemical Properties and Biological Activity	13
miRNA-Mediated Knockdown of ATXN3 Alleviates Molecular Disease Hallmarks in a Mouse Model for Spinocerebellar Ataxia Type 3	13
Biodistribution of Radioactively Labeled Splice Modulating Antisense Oligonucleotides After Intracerebroventricular and Intrathecal Injection in Mice	12
Allele-Selective LNA Gapmers for the Treatment of Fibrodysplasia Ossificans Progressiva Knock Down the Pathogenic ACVR1^R206H Transcript and Inhibit Osteogenic Differentiation	12
Hepatotoxicity Reduction Profiles of Antisense Oligonucleotides Containing Amido-Bridged Nucleic Acid and 2′-O,4′-C-Spirocyclopropylene Bridged Nucleic Acid	11
Opportunities for More Tailored Approaches to Genotoxicity Testing and Carcinogenicity Strategy for Oligonucleotide Therapeutics: Outcome of an Industry Survey	11
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
Addressing the Challenges of Treating Patients with Heterozygous Gain of Function Mutations	10
Preclinical Pharmacokinetics in Tumors and Normal Tissues of the Antigene PNA Oligonucleotide MYCN-Inhibitor BGA002	10
Binding and Ligand Activation Driven Enrichment–Directed Evolution of SaCas9 gRNAs Improves Gene Editing Efficiency	10
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 SOD1 Mutated Amyotr	9
Modulation of Gene Expression in the Eye with Antisense Oligonucleotides	9
Considerations for Creating the Next Generation of RNA Therapeutics: Oligonucleotide Chemistry and Innate Immune Responses to Nucleic Acids	9
Antisense Oligonucleotide-Based Rescue of Complex Intronic Splicing Defects in ABCA4	9
CRISPR/Cas and Hepatitis B Therapy: Technological Advances and Practical Barriers	9
Elevation of SHANK3 Levels by Antisense Oligonucleotides Directed Against the 3′-UTR of the Human SHANK3 mRNA	8
Cholesterol-Conjugated siRNA Silencing Tnf for the Treatment of Liver Macrophage-Mediated Acute Inflammation in Nonalcoholic Fatty Liver Disease	8
The Quest for mRNA Vaccines	8
Development of Long Asymmetric siRNA Structure for Target Gene Silencing and Immune Stimulation in Mammalian Cells	8
Addressing the Needs of Nano-Rare Patients	8
Multiplexed In Vivo Screening Using Barcoded Aptamer Technology to Identify Oligonucleotide-Based Targeting Reagents	7
Experimental Model Systems Used in the Preclinical Development of Nucleic Acid Therapeutics	7
Characterizing Antibodies Targeting Antisense Oligonucleotide Phosphorothioate and 2′- O -Methoxyethyl Modifications for Intracellular Trafficking and Bi	6
Impact of the Inhibition of Organic Anion Transporter on Tricyclo-DNA-Mediated Exon Skipping in the mdx Mouse Model	6
Efficient Downregulation of Alk4 in Skeletal Muscle After Systemic Treatment with Conjugated siRNAs in a Mouse Model for Duchenne Muscular Dystrophy	6
Acute Neurotoxicity of Antisense Oligonucleotides After Intracerebroventricular Injection Into Mouse Brain Can Be Predicted from Sequence Features	6
Rosalind Franklin Society Proudly Announces the 2022 Award Recipient for Nucleic Acid Therapeutics	6
Applying Synthetic Nucleic Acid Therapeutics to Gene Activation for Haploinsufficient Diseases	6
Preparing n-of-1 Antisense Oligonucleotide Treatments for Rare Neurological Diseases in Europe: Genetic, Regulatory, and Ethical Perspectives	6
Aerosolized Pulmonary Delivery of mRNA Constructs Attenuates Severity of Escherichia coli Pneumonia in the Rat	6
Time Is a Critical Factor When Evaluating Oligonucleotide Therapeutics in hERG Assays	5
A Workflow for Transcriptome-Wide Assessment of Antisense Oligonucleotide Selectivity	5
Immunogenicity Risk Assessment for Nucleic Acid Therapeutics: A Comprehensive Evaluation for ASO, siRNA, and Nonvaccine mRNA/LNP Therapies by the IQ Consortium	5
Antisense Oligonucleotide Quantification via Splint-Ligation PCR Assay in Nonhuman Primate Central Nervous System Tissues and Biofluids	5
mRNA Nuclear Clustering Leads to a Difference in Mutant Huntingtin mRNA and Protein Silencing by siRNAs In Vivo	5
Nucleic Acid Therapeutics: Successes, Milestones, and Upcoming Innovation	5
Altered Biodistribution and Hepatic Safety Profile of a Gapmer Antisense Oligonucleotide Bearing Guanidine-Bridged Nucleic Acids	5
Challenges and Opportunities for Nucleic Acid Therapeutics	5
Antisense Oligonucleotide STK-002 Increases OPA1 in Retina and Improves Mitochondrial Function in Autosomal Dominant Optic Atrophy Cells	4
Report of the European Medicines Agency Conference on RNA-Based Medicines	4

It is Time to Revisit miRNA Therapeutics	4
Host-Directed Targeting of LincRNA-MIR99AHG Suppresses Intracellular Growth of Mycobacterium tuberculosis	4
Quantification of Antisense Oligonucleotides by Splint Ligation and Quantitative Polymerase Chain Reaction	4