Nucleic Acid Therapeutics

Papers
(The TQCC of Nucleic Acid Therapeutics is 11. 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 2021-05-01 to 2025-05-01.)
ArticleCitations
SIDT2 Inhibits Phosphorothioate Antisense Oligonucleotide Activity by Regulating Cellular Localization of Lysosomes84
Oligonucleotides Targeting DNA Repeats Downregulate Huntingtin Gene Expression in Huntington's Patient-Derived Neural Model System75
Assessing Hybridization-Dependent Off-Target Risk for Therapeutic Oligonucleotides: Updated Industry Recommendations61
Combined 20-Hydroxyecdysone and Antisense-Mediated Exon Skipping Improve Functional Outcomes in a Mouse Model of Duchenne Muscular Dystrophy59
Knockdown of Circular RNAs Using LNA-Modified Antisense Oligonucleotides55
Mechanistic Insights into Hybridization-Based Off-Target Activity of GalNAc-siRNA Conjugates43
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 Dystrophy41
Therapeutic siRNA Loaded to RISC as Single and Double Strands Requires an Appropriate Quantitative Assay for RISC PK Assessment38
Screening Splice-Switching Antisense Oligonucleotides in Pancreas-Cancer Organoids37
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., Nucleic35
Combining Heparin and a FX/Xa Aptamer to Reduce Thrombin Generation in Cardiopulmonary Bypass and COVID-1935
Optimization of RNA Aptamer SELEX Methods: Improved Aptamer Transcript 3′-End Homogeneity, PAGE Purification Yield, and Target-Bound Aptamer RNA Recovery33
Inhibition of Epidermal Growth Factor Receptor Signaling by Antisense Oligonucleotides as a Novel Approach to Epidermal Growth Factor Receptor Inhibition32
Reversible Aptamer Staining, Sorting, and Cleaning of Cells (Clean FACS) with Antidote Oligonucleotide or Nuclease Yields Fully Responsive Cells31
Modulating Polymer-siRNA Binding Does Not Promote Polyplex-Mediated Silencing29
17th Annual Meeting of the Oligonucleotide Therapeutics Society: A Tribute to Bob Letsinger, Progress in N = 1 Treatments, and Successful First In-Humans CRISPR Trials25
Improvements to Hybridization-Ligation ELISA Methods to Overcome Bioanalytical Challenges Posed by Novel Oligonucleotide Therapeutics24
Considerations for the Terminal Sterilization of Oligonucleotide Drug Products23
Sequence- and Structure-Dependent Cytotoxicity of Phosphorothioate and 2′-O-Methyl Modified Single-Stranded Oligonucleotides22
Cross-Species Translation of Biophase Half-Life and Potency of GalNAc-Conjugated siRNAs20
Investigation of the Activity of Antisense Oligonucleotides Targeting Multiple Genes by RNA-Sequencing20
A Combined Fertility and Developmental Toxicity Study with an Antisense Oligonucleotide Targeting Murine Apolipoprotein C-III mRNA in Mice19
Near Sequence Homology Does Not Guarantee siRNA Cross-Species Efficacy18
Pharmacodynamic and Pharmacokinetic Properties of Full Phosphorothioate Small Interfering RNAs for Gene Silencing In Vivo18
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 Tr18
miR-21: A Key Small Molecule with Great Effects in Combination Cancer Therapy16
Individualized Antisense Oligonucleotide Therapies: How to Approach the Challenge of Manufacturing These Oligos from a Chemistry, Manufacturing, and Control-Regulatory Standpoint15
Aptamers Targeting Hallmark Proteins of Neurodegeneration13
Sequence-Controlled Spherical Nucleic Acids: Gene Silencing, Encapsulation, and Cellular Uptake13
RIG-I-Mediated Innate Immune Stimulation by Chemically Synthesized Long Double-Stranded RNAs Is Structure and Sequence Dependent13
DNAzymes: Expanding the Potential of Nucleic Acid Therapeutics13
miRNA-Mediated Knockdown of ATXN3 Alleviates Molecular Disease Hallmarks in a Mouse Model for Spinocerebellar Ataxia Type 312
Rapid and Reliable Quantification of Prime Editing Targeting Within the Porcine ABCA4 Gene Using a BRET-Based Sensor12
Peptide Nucleic Acid-Mediated Regulation of CRISPR-Cas9 Specificity12
RNA Interference Effectors Selectively Silence the Pathogenic Variant GNAO1 c.607 G > A In Vitro11
Biodistribution of Radioactively Labeled Splice Modulating Antisense Oligonucleotides After Intracerebroventricular and Intrathecal Injection in Mice11
Modification of KL4 Peptide Revealed the Importance of Alpha-Helical Structure for Efficient siRNA Delivery11
Allele-Selective LNA Gapmers for the Treatment of Fibrodysplasia Ossificans Progressiva Knock Down the Pathogenic ACVR1R206H Transcript and Inhibit Osteogenic Differentiation11
Splice-Switching Antisense Oligonucleotides Correct Phenylalanine Hydroxylase Exon 11 Skipping Defects and Rescue Enzyme Activity in Phenylketonuria11
Non-Small-Cell Lung Cancer Regression by siRNA Delivered Through Exosomes That Display EGFR RNA Aptamer11
Binary Antisense Oligonucleotide Agent for Cancer Marker-Dependent Degradation of Targeted RNA11
Levels of Exon-Skipping Are Not Artificially Overestimated Because of the Increased Affinity of Tricyclo-DNA-Modified Antisense Oligonucleotides to the Target DMD Exon11
New Oligonucleotide 2′-O-Alkyl N3′→P5′ (Thio)-Phosphoramidates as Potent Antisense Agents: Physicochemical Properties and Biological Activity11
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