Nucleic Acid Therapeutics

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 2020-05-01 to 2024-05-01.)
ArticleCitations
Efficiency and Target Derepression of Anti-miR-92a: Results of a First in Human Study96
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 Tr59
Transcriptome-Wide Off-Target Effects of Steric-Blocking Oligonucleotides45
Delivery of RNA Therapeutics: The Great Endosomal Escape!43
Preparing n-of-1 Antisense Oligonucleotide Treatments for Rare Neurological Diseases in Europe: Genetic, Regulatory, and Ethical Perspectives38
Folate Receptor-Mediated siRNA Delivery: Recent Developments and Future Directions for RNAi Therapeutics29
Challenges and Opportunities for Nucleic Acid Therapeutics28
Oligonucleotides and the COVID-19 Pandemic: A Perspective26
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-5p25
Non-Small-Cell Lung Cancer Regression by siRNA Delivered Through Exosomes That Display EGFR RNA Aptamer24
Technical Considerations for Use of Oligonucleotide Solution API22
Acute Neurotoxicity of Antisense Oligonucleotides After Intracerebroventricular Injection Into Mouse Brain Can Be Predicted from Sequence Features22
Likelihood of Nonspecific Activity of Gapmer Antisense Oligonucleotides Is Associated with Relative Hybridization Free Energy21
Researcher's Perceptions on Publishing “Negative” Results and Open Access20
Delivery of Antisense Oligonucleotides to the Cornea19
miR-21: A Key Small Molecule with Great Effects in Combination Cancer Therapy19
Mesyl Phosphoramidate Oligonucleotides as Potential Splice-Switching Agents: Impact of Backbone Structure on Activity and Intracellular Localization18
Antisense Oligonucleotides as Potential Therapeutics for Type 2 Diabetes17
Population Pharmacokinetic–Pharmacodynamic Modeling of Inotersen, an Antisense Oligonucleotide for Treatment of Patients with Hereditary Transthyretin Amyloidosis17
Drug Metabolism and Pharmacokinetics of Antisense Oligonucleotide Therapeutics: Typical Profiles, Evaluation Approaches, and Points to Consider Compared with Small Molecule Drugs17
Immunogenicity Assessment of Inotersen, a 2′-O-(2-Methoxyethyl) Antisense Oligonucleotide in Animals and Humans: Effect on Pharmacokinetics, Pharmacodynamics, and Safety16
Human Papillomavirus G-Rich Regions as Potential Antiviral Drug Targets15
Population Pharmacokinetic–Pharmacodynamic Model of Serum Transthyretin Following Patisiran Administration15
Considerations in the Preclinical Assessment of the Safety of Antisense Oligonucleotides15
Quantification of Antisense Oligonucleotides by Splint Ligation and Quantitative Polymerase Chain Reaction15
Pyrene-Modified DNA Aptamers with High Affinity to Wild-Type EGFR and EGFRvIII14
Assay, Purity, and Impurity Profile of Phosphorothioate Oligonucleotide Therapeutics by Ion Pair–HPLC–MS13
Effects of Chemical Modifications on siRNA Strand Selection in Mammalian Cells12
Perspectives on the Designation of Oligonucleotide Starting Materials12
Anti-CD44 DNA Aptamers Selectively Target Cancer Cells12
Impact of Serum Proteins on the Uptake and RNAi Activity of GalNAc-Conjugated siRNAs12
RT-qPCR Methods to Support Pharmacokinetics and Drug Mechanism of Action to Advance Development of RNAi Therapeutics11
Interspecies Scaling of Human Clearance and Plasma Trough Exposure for Antisense Oligonucleotides: A Retrospective Analysis of GalNAc3-Conjugated and Unconjugated-Antisense Oligonucleotides11
Knockdown of Muscle-Specific Ribosomal Protein L3-Like Enhances Muscle Function in Healthy and Dystrophic Mice11
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-ASOs11
The Performance of Minicircle DNA Versus Parental Plasmid in p53 Gene Delivery Into HPV-18-Infected Cervical Cancer Cells11
Modulation of RNA Splicing by Oligonucleotides: Mechanisms of Action and Therapeutic Implications11
Modification of KL4 Peptide Revealed the Importance of Alpha-Helical Structure for Efficient siRNA Delivery11
Consensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N-of-1 Exon Skipping Antisense Oligonucleotides11
Pharmacodynamic and Pharmacokinetic Properties of Full Phosphorothioate Small Interfering RNAs for Gene Silencing In Vivo10
Solid-Phase Separation of Toxic Phosphorothioate Antisense Oligonucleotide-Protein Nucleolar Aggregates Is Cytoprotective10
Strategies for Identity Testing of Therapeutic Oligonucleotide Drug Substances and Drug Products10
Identification and Optimization of a Minor Allele-Specific siRNA to Prevent PNPLA3 I148M-Driven Nonalcoholic Fatty Liver Disease10
Evaluation of Phosphorus and Non-Phosphorus Neutral Oligonucleotide Backbones for Enhancing Therapeutic Index of Gapmer Antisense Oligonucleotides10
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