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 2020-11-01 to 2024-11-01.)

Article	Citations
Efficiency and Target Derepression of Anti-miR-92a: Results of a First in Human Study	102
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	66
Delivery of RNA Therapeutics: The Great Endosomal Escape!	56
Transcriptome-Wide Off-Target Effects of Steric-Blocking Oligonucleotides	51
Preparing n-of-1 Antisense Oligonucleotide Treatments for Rare Neurological Diseases in Europe: Genetic, Regulatory, and Ethical Perspectives	49
Challenges and Opportunities for Nucleic Acid Therapeutics	34
Drug Metabolism and Pharmacokinetics of Antisense Oligonucleotide Therapeutics: Typical Profiles, Evaluation Approaches, and Points to Consider Compared with Small Molecule Drugs	33
Folate Receptor-Mediated siRNA Delivery: Recent Developments and Future Directions for RNAi Therapeutics	30
Non-Small-Cell Lung Cancer Regression by siRNA Delivered Through Exosomes That Display EGFR RNA Aptamer	29
Acute Neurotoxicity of Antisense Oligonucleotides After Intracerebroventricular Injection Into Mouse Brain Can Be Predicted from Sequence Features	27
Considerations in the Preclinical Assessment of the Safety of Antisense Oligonucleotides	25
Researcher's Perceptions on Publishing “Negative” Results and Open Access	23
miR-21: A Key Small Molecule with Great Effects in Combination Cancer Therapy	21
Consensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N-of-1 Exon Skipping Antisense Oligonucleotides	20
Antisense Oligonucleotides as Potential Therapeutics for Type 2 Diabetes	20
Quantification of Antisense Oligonucleotides by Splint Ligation and Quantitative Polymerase Chain Reaction	18
Mesyl Phosphoramidate Oligonucleotides as Potential Splice-Switching Agents: Impact of Backbone Structure on Activity and Intracellular Localization	18
Human Papillomavirus G-Rich Regions as Potential Antiviral Drug Targets	17
Assay, Purity, and Impurity Profile of Phosphorothioate Oligonucleotide Therapeutics by Ion Pair–HPLC–MS	17
Translational Pharmacokinetic/Pharmacodynamic Model for mRNA-3927, an Investigational Therapeutic for the Treatment of Propionic Acidemia	16
Modulation of RNA Splicing by Oligonucleotides: Mechanisms of Action and Therapeutic Implications	16
Addressing the Needs of Patients with Ultra-Rare Mutations One Patient at a Time: The n-Lorem Approach	13
Impact of Serum Proteins on the Uptake and RNAi Activity of GalNAc-Conjugated siRNAs	13
Perspectives on the Designation of Oligonucleotide Starting Materials	13
DNAzymes: Expanding the Potential of Nucleic Acid Therapeutics	13

The Combination of Mesyl-Phosphoramidate Inter-Nucleotide Linkages and 2′-O-Methyl in Selected Positions in the Antisense Oligonucleotide Enhances the Performance of RNaseH1 Active PS-ASOs	12
Interspecies Scaling of Human Clearance and Plasma Trough Exposure for Antisense Oligonucleotides: A Retrospective Analysis of GalNAc3-Conjugated and Unconjugated-Antisense Oligonucleotides	12
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	12
Inhomogeneous Diastereomeric Composition of Mongersen Antisense Phosphorothioate Oligonucleotide Preparations and Related Pharmacological Activity Impairment	12
The Performance of Minicircle DNA Versus Parental Plasmid in p53 Gene Delivery Into HPV-18-Infected Cervical Cancer Cells	12
Evaluation of Phosphorus and Non-Phosphorus Neutral Oligonucleotide Backbones for Enhancing Therapeutic Index of Gapmer Antisense Oligonucleotides	11
Modification of KL4 Peptide Revealed the Importance of Alpha-Helical Structure for Efficient siRNA Delivery	11
Chitosan-Shelled Nanobubbles Irreversibly Encapsulate Morpholino Conjugate Antisense Oligonucleotides and Are Ineffective for Phosphorodiamidate Morpholino-Mediated Gene Silencing of DUX4	11
Knockdown of Muscle-Specific Ribosomal Protein L3-Like Enhances Muscle Function in Healthy and Dystrophic Mice	11
Improved Anti-Prion Nucleic Acid Aptamers by Incorporation of Chemical Modifications	11
Solid-Phase Separation of Toxic Phosphorothioate Antisense Oligonucleotide-Protein Nucleolar Aggregates Is Cytoprotective	11
Identification and Optimization of a Minor Allele-Specific siRNA to Prevent PNPLA3 I148M-Driven Nonalcoholic Fatty Liver Disease	11
miRNA-Mediated Knockdown of ATXN3 Alleviates Molecular Disease Hallmarks in a Mouse Model for Spinocerebellar Ataxia Type 3	10
Antisense Oligonucleotide-Induced Amyloid Precursor Protein Splicing Modulation as a Therapeutic Approach for Dutch-Type Cerebral Amyloid Angiopathy	10
Pharmacodynamic and Pharmacokinetic Properties of Full Phosphorothioate Small Interfering RNAs for Gene Silencing In Vivo	10
Early-Stage Identification and Avoidance of Antisense Oligonucleotides Causing Species-Specific Inflammatory Responses in Human Volunteer Peripheral Blood Mononuclear Cells	9
Antisense Oligonucleotide Rescue of Deep-Intronic Variants Activating Pseudoexons in the 6-Pyruvoyl-Tetrahydropterin Synthase Gene	9
Individualized Therapeutics Development for Rare Diseases: The Current Ethical Landscape and Policy Responses	9
Immortalized Canine Dystrophic Myoblast Cell Lines for Development of Peptide-Conjugated Splice-Switching Oligonucleotides	9
Short-Chain Guide RNA for Site-Directed A-to-I RNA Editing	8
Host-Directed Targeting of LincRNA-MIR99AHG Suppresses Intracellular Growth of Mycobacterium tuberculosis	8
Enhancing SIRT1 Gene Expression Using Small Activating RNAs: A Novel Approach for Reversing Metabolic Syndrome	8
Delivery Characterization of SPL84 Inhaled Antisense Oligonucleotide Drug for 3849 + 10 kb C- > T Cystic Fibrosis Patients	8
Development of Human CBF1-Targeting Single-Stranded DNA Aptamers with Antiangiogenic Activity In Vitro	8
Differential Uptake of Antisense Oligonucleotides in Mouse Hepatocytes and Macrophages Revealed by Simultaneous Two-Photon Excited Fluorescence and Coherent Raman Imaging	8
Assessment of the Immunogenicity Potential for Oligonucleotide-Based Drugs	7
Outcomes of the European Federation of Pharmaceutical Industries and Associations Oligonucleotide Working Group Survey on Nonclinical Practices and Regulatory Expectations for Therapeutic Oligonucleot	7
Considerations of the Japanese Research Working Group for the ICH S6 & Related Issues Regarding Nonclinical Safety Assessments of Oligonucleotide Therapeutics: Comparison with Those of Biopharmace	7
Optimization of RNA Aptamer SELEX Methods: Improved Aptamer Transcript 3′-End Homogeneity, PAGE Purification Yield, and Target-Bound Aptamer RNA Recovery	7
Improvements in the Tolerability Profile of 2′-O-Methoxyethyl Chimeric Antisense Oligonucleotides in Parallel with Advances in Design, Screening, and Other Methods	7
Altered Biodistribution and Hepatic Safety Profile of a Gapmer Antisense Oligonucleotide Bearing Guanidine-Bridged Nucleic Acids	7
Exon Skipping Through Chimeric Antisense U1 snRNAs to Correct Retinitis Pigmentosa GTPase-Regulator (RPGR) Splice Defect	7
Efficient Downregulation of Alk4 in Skeletal Muscle After Systemic Treatment with Conjugated siRNAs in a Mouse Model for Duchenne Muscular Dystrophy	7
Integrated Assessment of Phase 2 Data on GalNAc₃-Conjugated 2′-O-Methoxyethyl-Modified Antisense Oligonucleotides	7
Cross-Species Translation of Biophase Half-Life and Potency of GalNAc-Conjugated siRNAs	7
Elimination of Off-Target Effect by Chemical Modification of 5′-End of siRNA	7
Approaches for Systemic Delivery of Dystrophin Antisense Peptide Nucleic Acid in the mdx Mouse Model	7
Systematic Analysis of Chemical Modifications of Phosphorothioate Antisense Oligonucleotides that Modulate Their Innate Immune Response	7
To Conjugate or to Package? A Look at Targeted siRNA Delivery Through Folate Receptors	6
Development of Prodrug Type Circular siRNA for In Vivo Knockdown by Systemic Administration	6
Binary Antisense Oligonucleotide Agent for Cancer Marker-Dependent Degradation of Targeted RNA	6
Sequence-Controlled Spherical Nucleic Acids: Gene Silencing, Encapsulation, and Cellular Uptake	6
Characterization of In Vitro G-Quadruplex Formation of Imetelstat Telomerase Inhibitor	6
Inflammatory Non-CpG Antisense Oligonucleotides Are Signaling Through TLR9 in Human Burkitt Lymphoma B Bjab Cells	5
Next Generation Exon 51 Skipping Antisense Oligonucleotides for Duchenne Muscular Dystrophy	5
Programmed Instability of Ligand Conjugation Manifold for Efficient Hepatocyte Delivery of Therapeutic Oligonucleotides	5
Targeted Delivery of Antisense Oligonucleotides Through Angiotensin Type 1 Receptor	5
Hsc70 Facilitates Mannose-6-Phosphate Receptor-Mediated Intracellular Trafficking and Enhances Endosomal Release of Phosphorothioate-Modified Antisense Oligonucleotides	5
A Novel DNA Aptamer Targeting S100P Induces Antitumor Effects in Colorectal Cancer Cells	4
Oligonucleotide IMT504 Improves Glucose Metabolism and Controls Immune Cell Mediators in Female Diabetic NOD Mice	4

Individualized Antisense Oligonucleotide Therapies: How to Approach the Challenge of Manufacturing These Oligos from a Chemistry, Manufacturing, and Control-Regulatory Standpoint	4
Oligonucleotides Targeting DNA Repeats Downregulate Huntingtin Gene Expression in Huntington's Patient-Derived Neural Model System	4
CRISPR/Cas and Hepatitis B Therapy: Technological Advances and Practical Barriers	4
Evaluation of Pharmacokinetics and Metabolism of Phosphorothioate Antisense Oligonucleotide G3139 in Rat by Capillary Electrophoresis with Laser-Induced Fluorescence	4
Allele-Selective LNA Gapmers for the Treatment of Fibrodysplasia Ossificans Progressiva Knock Down the Pathogenic ACVR1^R206H Transcript and Inhibit Osteogenic Differentiation	4
Aptamers Targeting Hallmark Proteins of Neurodegeneration	4
Rapid and Reliable Quantification of Prime Editing Targeting Within the Porcine ABCA4 Gene Using a BRET-Based Sensor	4
An Integrative Multi-Omics Analysis Reveals MicroRNA-143 as a Potential Therapeutic to Attenuate Retinal Angiogenesis	4
Nucleic Acid Therapeutics: Successes, Milestones, and Upcoming Innovation	4
Improvements to Hybridization-Ligation ELISA Methods to Overcome Bioanalytical Challenges Posed by Novel Oligonucleotide Therapeutics	4
Aerosolized Pulmonary Delivery of mRNA Constructs Attenuates Severity of Escherichia coli Pneumonia in the Rat	4
Delivery of Antisense DNA into Pathogenic Parasite Trypanosoma cruzi Using Virus-Like Protein-Based Nanoparticles	4
Knockdown of Circular RNAs Using LNA-Modified Antisense Oligonucleotides	4
2′-O-(N-(Aminoethyl)carbamoyl)methyl Modification Allows for Lower Phosphorothioate Content in Splice-Switching Oligonucleotides with Retained Activity	4