OOIR: Observatory of International Research

Papers

(The TQCC of Nucleic Acid Therapeutics is 12. 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 2021-08-01 to 2025-08-01.)

Article	Citations
Knockdown of Circular RNAs Using LNA-Modified Antisense Oligonucleotides	94
Combined 20-Hydroxyecdysone and Antisense-Mediated Exon Skipping Improve Functional Outcomes in a Mouse Model of Duchenne Muscular Dystrophy	87
Oligonucleotides Targeting DNA Repeats Downregulate Huntingtin Gene Expression in Huntington's Patient-Derived Neural Model System	70
SIDT2 Inhibits Phosphorothioate Antisense Oligonucleotide Activity by Regulating Cellular Localization of Lysosomes	61
Assessing Hybridization-Dependent Off-Target Risk for Therapeutic Oligonucleotides: Updated Industry Recommendations	60
Mechanistic Insights into Hybridization-Based Off-Target Activity of GalNAc-siRNA Conjugates	55
Combining Heparin and a FX/Xa Aptamer to Reduce Thrombin Generation in Cardiopulmonary Bypass and COVID-19	49
Therapeutic siRNA Loaded to RISC as Single and Double Strands Requires an Appropriate Quantitative Assay for RISC PK Assessment	47
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	39
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	38
Screening Splice-Switching Antisense Oligonucleotides in Pancreas-Cancer Organoids	37
Reversible Aptamer Staining, Sorting, and Cleaning of Cells (Clean FACS) with Antidote Oligonucleotide or Nuclease Yields Fully Responsive Cells	36
Inhibition of Epidermal Growth Factor Receptor Signaling by Antisense Oligonucleotides as a Novel Approach to Epidermal Growth Factor Receptor Inhibition	35
Design and In Vitro Evaluation of a Novel Antisense Oligonucleotide for Treatment of BK Virus Infection	35
Optimization of RNA Aptamer SELEX Methods: Improved Aptamer Transcript 3′-End Homogeneity, PAGE Purification Yield, and Target-Bound Aptamer RNA Recovery	31
Improvements to Hybridization-Ligation ELISA Methods to Overcome Bioanalytical Challenges Posed by Novel Oligonucleotide Therapeutics	27
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	26
Considerations for the Terminal Sterilization of Oligonucleotide Drug Products	25
Sequence- and Structure-Dependent Cytotoxicity of Phosphorothioate and 2′-O-Methyl Modified Single-Stranded Oligonucleotides	23
Cross-Species Translation of Biophase Half-Life and Potency of GalNAc-Conjugated siRNAs	23
A Combined Fertility and Developmental Toxicity Study with an Antisense Oligonucleotide Targeting Murine Apolipoprotein C-III mRNA in Mice	21
Near Sequence Homology Does Not Guarantee siRNA Cross-Species Efficacy	20
Investigation of the Activity of Antisense Oligonucleotides Targeting Multiple Genes by RNA-Sequencing	18
miR-21: A Key Small Molecule with Great Effects in Combination Cancer Therapy	17
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	15

Individualized Antisense Oligonucleotide Therapies: How to Approach the Challenge of Manufacturing These Oligos from a Chemistry, Manufacturing, and Control-Regulatory Standpoint	15
Rapid and Reliable Quantification of Prime Editing Targeting Within the Porcine ABCA4 Gene Using a BRET-Based Sensor	14
RIG-I-Mediated Innate Immune Stimulation by Chemically Synthesized Long Double-Stranded RNAs Is Structure and Sequence Dependent	14
Aptamers Targeting Hallmark Proteins of Neurodegeneration	14
Sequence-Controlled Spherical Nucleic Acids: Gene Silencing, Encapsulation, and Cellular Uptake	14
RNA Interference Effectors Selectively Silence the Pathogenic Variant GNAO1 c.607 G > A In Vitro	13
DNAzymes: Expanding the Potential of Nucleic Acid Therapeutics	13
miRNA-Mediated Knockdown of ATXN3 Alleviates Molecular Disease Hallmarks in a Mouse Model for Spinocerebellar Ataxia Type 3	13
Peptide Nucleic Acid-Mediated Regulation of CRISPR-Cas9 Specificity	13
Binary Antisense Oligonucleotide Agent for Cancer Marker-Dependent Degradation of Targeted RNA	13
Hepatotoxicity Reduction Profiles of Antisense Oligonucleotides Containing Amido-Bridged Nucleic Acid and 2′-O,4′-C-Spirocyclopropylene Bridged Nucleic Acid	12
New Oligonucleotide 2′-O-Alkyl N3′→P5′ (Thio)-Phosphoramidates as Potent Antisense Agents: Physicochemical Properties and Biological Activity	12
Allele-Selective LNA Gapmers for the Treatment of Fibrodysplasia Ossificans Progressiva Knock Down the Pathogenic ACVR1^R206H Transcript and Inhibit Osteogenic Differentiation	12
Levels of Exon-Skipping Are Not Artificially Overestimated Because of the Increased Affinity of Tricyclo-DNA-Modified Antisense Oligonucleotides to the Target DMD Exon	12
Opportunities for More Tailored Approaches to Genotoxicity Testing and Carcinogenicity Strategy for Oligonucleotide Therapeutics: Outcome of an Industry Survey	12
Biodistribution of Radioactively Labeled Splice Modulating Antisense Oligonucleotides After Intracerebroventricular and Intrathecal Injection in Mice	12