OOIR: Observatory of International Research

Papers

(The median citation count of Nucleic Acid Therapeutics is 5. 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 2020-03-01 to 2024-03-01.)

Article	Citations
Efficiency and Target Derepression of Anti-miR-92a: Results of a First in Human Study	89
The 10th Oligonucleotide Therapy Approved: Golodirsen for Duchenne Muscular Dystrophy	81
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	55
Transcriptome-Wide Off-Target Effects of Steric-Blocking Oligonucleotides	41
Bifunctional Aptamer–Doxorubicin Conjugate Crosses the Blood–Brain Barrier and Selectively Delivers Its Payload to EpCAM-Positive Tumor Cells	41
Preparing n-of-1 Antisense Oligonucleotide Treatments for Rare Neurological Diseases in Europe: Genetic, Regulatory, and Ethical Perspectives	33
Delivery of RNA Therapeutics: The Great Endosomal Escape!	31
Folate Receptor-Mediated siRNA Delivery: Recent Developments and Future Directions for RNAi Therapeutics	29
Challenges and Opportunities for Nucleic Acid Therapeutics	28
Oligonucleotides and the COVID-19 Pandemic: A Perspective	26
High Levels of Apoptosis Are Induced in the Human Colon Cancer HT-29 Cell Line by Co-Administration of Sulforaphane and a Peptide Nucleic Acid Targeting miR-15b-5p	25
Non-Small-Cell Lung Cancer Regression by siRNA Delivered Through Exosomes That Display EGFR RNA Aptamer	23
Underlying Immune Disorder May Predispose Some Transthyretin Amyloidosis Subjects to Inotersen-Mediated Thrombocytopenia	22
Likelihood of Nonspecific Activity of Gapmer Antisense Oligonucleotides Is Associated with Relative Hybridization Free Energy	21
Technical Considerations for Use of Oligonucleotide Solution API	20
Researcher's Perceptions on Publishing “Negative” Results and Open Access	19
Acute Neurotoxicity of Antisense Oligonucleotides After Intracerebroventricular Injection Into Mouse Brain Can Be Predicted from Sequence Features	19
miR-21: A Key Small Molecule with Great Effects in Combination Cancer Therapy	19
Delivery of Antisense Oligonucleotides to the Cornea	19
Mesyl Phosphoramidate Oligonucleotides as Potential Splice-Switching Agents: Impact of Backbone Structure on Activity and Intracellular Localization	17
Metabolic Benefits of MicroRNA-22 Inhibition	16
Drug Metabolism and Pharmacokinetics of Antisense Oligonucleotide Therapeutics: Typical Profiles, Evaluation Approaches, and Points to Consider Compared with Small Molecule Drugs	16
Population Pharmacokinetic–Pharmacodynamic Modeling of Inotersen, an Antisense Oligonucleotide for Treatment of Patients with Hereditary Transthyretin Amyloidosis	15
Human Papillomavirus G-Rich Regions as Potential Antiviral Drug Targets	15
Antisense Oligonucleotides as Potential Therapeutics for Type 2 Diabetes	14

Immunogenicity Assessment of Inotersen, a 2′-O-(2-Methoxyethyl) Antisense Oligonucleotide in Animals and Humans: Effect on Pharmacokinetics, Pharmacodynamics, and Safety	14
Population Pharmacokinetic–Pharmacodynamic Model of Serum Transthyretin Following Patisiran Administration	13
Pyrene-Modified DNA Aptamers with High Affinity to Wild-Type EGFR and EGFRvIII	13
Assay, Purity, and Impurity Profile of Phosphorothioate Oligonucleotide Therapeutics by Ion Pair–HPLC–MS	12
Anti-CD44 DNA Aptamers Selectively Target Cancer Cells	12
Quantification of Antisense Oligonucleotides by Splint Ligation and Quantitative Polymerase Chain Reaction	12
Effects of Chemical Modifications on siRNA Strand Selection in Mammalian Cells	12
Modification of KL4 Peptide Revealed the Importance of Alpha-Helical Structure for Efficient siRNA Delivery	11
Impact of Serum Proteins on the Uptake and RNAi Activity of GalNAc-Conjugated siRNAs	11
Interspecies Scaling of Human Clearance and Plasma Trough Exposure for Antisense Oligonucleotides: A Retrospective Analysis of GalNAc3-Conjugated and Unconjugated-Antisense Oligonucleotides	11
Considerations in the Preclinical Assessment of the Safety of Antisense Oligonucleotides	11
The Performance of Minicircle DNA Versus Parental Plasmid in p53 Gene Delivery Into HPV-18-Infected Cervical Cancer Cells	11
Knockdown of Muscle-Specific Ribosomal Protein L3-Like Enhances Muscle Function in Healthy and Dystrophic Mice	10
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	10
Perspectives on the Designation of Oligonucleotide Starting Materials	10
Modulation of RNA Splicing by Oligonucleotides: Mechanisms of Action and Therapeutic Implications	10
RT-qPCR Methods to Support Pharmacokinetics and Drug Mechanism of Action to Advance Development of RNAi Therapeutics	10
Solid-Phase Separation of Toxic Phosphorothioate Antisense Oligonucleotide-Protein Nucleolar Aggregates Is Cytoprotective	10
Pharmacodynamic and Pharmacokinetic Properties of Full Phosphorothioate Small Interfering RNAs for Gene Silencing In Vivo	10
Evaluation of Phosphorus and Non-Phosphorus Neutral Oligonucleotide Backbones for Enhancing Therapeutic Index of Gapmer Antisense Oligonucleotides	10
Chitosan-Shelled Nanobubbles Irreversibly Encapsulate Morpholino Conjugate Antisense Oligonucleotides and Are Ineffective for Phosphorodiamidate Morpholino-Mediated Gene Silencing of DUX4	9
Identification and Optimization of a Minor Allele-Specific siRNA to Prevent PNPLA3 I148M-Driven Nonalcoholic Fatty Liver Disease	9
Translational Pharmacokinetic/Pharmacodynamic Model for mRNA-3927, an Investigational Therapeutic for the Treatment of Propionic Acidemia	9
Novel MicroRNA Sponges to Specifically Modulate Gene Expression in Colon Cancer Cells	9
MYCN Silencing by RNAi Induces Neurogenesis and Suppresses Proliferation in Models of Neuroblastoma with Resistance to Retinoic Acid	9
Strategies for Identity Testing of Therapeutic Oligonucleotide Drug Substances and Drug Products	9
Immortalized Canine Dystrophic Myoblast Cell Lines for Development of Peptide-Conjugated Splice-Switching Oligonucleotides	9
Selective Modulation of the Protease Activated Protein C Using Exosite Inhibiting Aptamers	8
Addressing the Needs of Patients with Ultra-Rare Mutations One Patient at a Time: The n-Lorem Approach	8
Systemic Evaluation of Chimeric LNA/2′-O-Methyl Steric Blockers for Myotonic Dystrophy Type 1 Therapy	8
Development of Human CBF1-Targeting Single-Stranded DNA Aptamers with Antiangiogenic Activity In Vitro	8
Consensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N-of-1 Exon Skipping Antisense Oligonucleotides	8
Improved Anti-Prion Nucleic Acid Aptamers by Incorporation of Chemical Modifications	8
Antisense Oligonucleotide-Induced Amyloid Precursor Protein Splicing Modulation as a Therapeutic Approach for Dutch-Type Cerebral Amyloid Angiopathy	8
Enhancing SIRT1 Gene Expression Using Small Activating RNAs: A Novel Approach for Reversing Metabolic Syndrome	7
Short-Chain Guide RNA for Site-Directed A-to-I RNA Editing	7
Individualized Therapeutics Development for Rare Diseases: The Current Ethical Landscape and Policy Responses	7
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	7
Efficient Downregulation of Alk4 in Skeletal Muscle After Systemic Treatment with Conjugated siRNAs in a Mouse Model for Duchenne Muscular Dystrophy	7
Inhomogeneous Diastereomeric Composition of Mongersen Antisense Phosphorothioate Oligonucleotide Preparations and Related Pharmacological Activity Impairment	7
Approaches for Systemic Delivery of Dystrophin Antisense Peptide Nucleic Acid in the mdx Mouse Model	7
Improvements in the Tolerability Profile of 2′-O-Methoxyethyl Chimeric Antisense Oligonucleotides in Parallel with Advances in Design, Screening, and Other Methods	7
Early-Stage Identification and Avoidance of Antisense Oligonucleotides Causing Species-Specific Inflammatory Responses in Human Volunteer Peripheral Blood Mononuclear Cells	7
Characterization of In Vitro G-Quadruplex Formation of Imetelstat Telomerase Inhibitor	6
Elimination of Off-Target Effect by Chemical Modification of 5′-End of siRNA	6
Exon Skipping Through Chimeric Antisense U1 snRNAs to Correct Retinitis Pigmentosa GTPase-Regulator (RPGR) Splice Defect	6
Differential Uptake of Antisense Oligonucleotides in Mouse Hepatocytes and Macrophages Revealed by Simultaneous Two-Photon Excited Fluorescence and Coherent Raman Imaging	6
miRNA-Mediated Knockdown of ATXN3 Alleviates Molecular Disease Hallmarks in a Mouse Model for Spinocerebellar Ataxia Type 3	6
Altered Biodistribution and Hepatic Safety Profile of a Gapmer Antisense Oligonucleotide Bearing Guanidine-Bridged Nucleic Acids	6
Gapmer Antisense Oligonucleotides Targeting 5S Ribosomal RNA Can Reduce Mature 5S Ribosomal RNA by Two Mechanisms	6

Development of Prodrug Type Circular siRNA for In Vivo Knockdown by Systemic Administration	6
DNAzymes: Expanding the Potential of Nucleic Acid Therapeutics	6
Outcomes of the European Federation of Pharmaceutical Industries and Associations Oligonucleotide Working Group Survey on Nonclinical Practices and Regulatory Expectations for Therapeutic Oligonucleot	6
Antisense Oligonucleotide Rescue of Deep-Intronic Variants Activating Pseudoexons in the 6-Pyruvoyl-Tetrahydropterin Synthase Gene	6
Cross-Species Translation of Biophase Half-Life and Potency of GalNAc-Conjugated siRNAs	5
Optimization of RNA Aptamer SELEX Methods: Improved Aptamer Transcript 3′-End Homogeneity, PAGE Purification Yield, and Target-Bound Aptamer RNA Recovery	5
Assessment of the Immunogenicity Potential for Oligonucleotide-Based Drugs	5
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	5
To Conjugate or to Package? A Look at Targeted siRNA Delivery Through Folate Receptors	5
Sequence-Controlled Spherical Nucleic Acids: Gene Silencing, Encapsulation, and Cellular Uptake	5
Systematic Analysis of Chemical Modifications of Phosphorothioate Antisense Oligonucleotides that Modulate Their Innate Immune Response	5
Integrated Assessment of Phase 2 Data on GalNAc₃-Conjugated 2′-O-Methoxyethyl-Modified Antisense Oligonucleotides	5
Host-Directed Targeting of LincRNA-MIR99AHG Suppresses Intracellular Growth of Mycobacterium tuberculosis	5