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 2021-04-01 to 2025-04-01.)

Article	Citations
It is Time to Revisit miRNA Therapeutics	83
Assessing Hybridization-Dependent Off-Target Risk for Therapeutic Oligonucleotides: Updated Industry Recommendations	72
Bioanalytical Assays for Oligonucleotide Therapeutics: Adding Antibody-Based Immunoassays to the Toolbox as an Orthogonal Approach to LC-MS/MS and Ligand Binding Assays	60
DNAzymes: Expanding the Potential of Nucleic Acid Therapeutics	58
SIDT2 Inhibits Phosphorothioate Antisense Oligonucleotide Activity by Regulating Cellular Localization of Lysosomes	53
Oligonucleotides Targeting DNA Repeats Downregulate Huntingtin Gene Expression in Huntington's Patient-Derived Neural Model System	42
Addressing the Needs of Nano-Rare Patients	38
Individualized Therapeutics Development for Rare Diseases: The Current Ethical Landscape and Policy Responses	36
What Do COVID-19 Vaccines Tell Us About Nucleic Acid Delivery In Vivo?	35
Individualized Antisense Oligonucleotide Therapies: How to Approach the Challenge of Manufacturing These Oligos from a Chemistry, Manufacturing, and Control-Regulatory Standpoint	34
RIG-I-Mediated Innate Immune Stimulation by Chemically Synthesized Long Double-Stranded RNAs Is Structure and Sequence Dependent	31
Hsc70 Facilitates Mannose-6-Phosphate Receptor-Mediated Intracellular Trafficking and Enhances Endosomal Release of Phosphorothioate-Modified Antisense Oligonucleotides	30
Combined 20-Hydroxyecdysone and Antisense-Mediated Exon Skipping Improve Functional Outcomes in a Mouse Model of Duchenne Muscular Dystrophy	30
Highly Potent Antisense Oligonucleotides Locked Nucleic Acid Gapmers Targeting the SARS-CoV-2 RNA Genome	30
Antisense Oligonucleotide-Mediated Silencing of Mitochondrial Fusion and Fission Factors Modulates Mitochondrial Dynamics and Rescues Mitochondrial Dysfunction	28
Inhomogeneous Diastereomeric Composition of Mongersen Antisense Phosphorothioate Oligonucleotide Preparations and Related Pharmacological Activity Impairment	23
Antisense Oligonucleotide-Based Rescue of Complex Intronic Splicing Defects in ABCA4	23
Host-Directed Targeting of LincRNA-MIR99AHG Suppresses Intracellular Growth of Mycobacterium tuberculosis	22
Assessment of the Immunogenicity Potential for Oligonucleotide-Based Drugs	20
Aptamers Targeting Hallmark Proteins of Neurodegeneration	19
Characterization of the TLR9-Activating Potential of LNA-Modified Antisense Oligonucleotides	18
Antisense Oligonucleotide STK-002 Increases OPA1 in Retina and Improves Mitochondrial Function in Autosomal Dominant Optic Atrophy Cells	18
Integrated Assessment of Phase 2 Data on GalNAc₃-Conjugated 2′-O-Methoxyethyl-Modified Antisense Oligonucleotides	17
Knockdown of Circular RNAs Using LNA-Modified Antisense Oligonucleotides	16
Considerations for Creating the Next Generation of RNA Therapeutics: Oligonucleotide Chemistry and Innate Immune Responses to Nucleic Acids	13

Sequence-Controlled Spherical Nucleic Acids: Gene Silencing, Encapsulation, and Cellular Uptake	13
Antisense Oligonucleotide-Induced Amyloid Precursor Protein Splicing Modulation as a Therapeutic Approach for Dutch-Type Cerebral Amyloid Angiopathy	13
Modulation of Gene Expression in the Eye with Antisense Oligonucleotides	13
Rapid and Reliable Quantification of Prime Editing Targeting Within the Porcine ABCA4 Gene Using a BRET-Based Sensor	13
Quantification of Antisense Oligonucleotides by Splint Ligation and Quantitative Polymerase Chain Reaction	13
Early-Stage Identification and Avoidance of Antisense Oligonucleotides Causing Species-Specific Inflammatory Responses in Human Volunteer Peripheral Blood Mononuclear Cells	13
Identification and Optimization of a Minor Allele-Specific siRNA to Prevent PNPLA3 I148M-Driven Nonalcoholic Fatty Liver Disease	12
Modulation of RNA Splicing by Oligonucleotides: Mechanisms of Action and Therapeutic Implications	12
Experimental Model Systems Used in the Preclinical Development of Nucleic Acid Therapeutics	11
Peptide Nucleic Acid-Mediated Regulation of CRISPR-Cas9 Specificity	11
Safety and Tolerability of GalNAc₃-Conjugated Antisense Drugs Compared to the Same-Sequence 2′-O-Methoxyethyl-Modified Antisense Drugs: Results from an Integrated Assessment of Phase	11
Impact of Serum Proteins on the Uptake and RNAi Activity of GalNAc-Conjugated siRNAs	11
Understanding and Rescuing the Splicing Defect Caused by the Frequent ABCA4 Variant c.4253+43G>A Underlying Stargardt Disease	11
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	11
Levels of Exon-Skipping Are Not Artificially Overestimated Because of the Increased Affinity of Tricyclo-DNA-Modified Antisense Oligonucleotides to the Target DMD Exon	11
Cholesterol-Conjugated siRNA Silencing Tnf for the Treatment of Liver Macrophage-Mediated Acute Inflammation in Nonalcoholic Fatty Liver Disease	10
Positive Allosteric Modulation of Antithrombin's Inhibitory Activity by RNA Aptamers	10
Programmed Instability of Ligand Conjugation Manifold for Efficient Hepatocyte Delivery of Therapeutic Oligonucleotides	10
Development of Long Asymmetric siRNA Structure for Target Gene Silencing and Immune Stimulation in Mammalian Cells	10