| Lipid Nanoparticles Enabling Gene Therapies: From Concepts to Clinical Utility |
51 |
| GalNAc-siRNA Conjugates: Leading the Way for Delivery of RNAi Therapeutics |
37 |
| Journey of siRNA: Clinical Developments and Targeted Delivery |
25 |
| Current Challenges in Delivery and Cytosolic Translocation of Therapeutic RNAs |
22 |
| Intracellular Trafficking and Endosomal Release of Oligonucleotides: What We Know and What We Do Not |
20 |
| Improving Small Interfering RNA Delivery In Vivo Through Lipid Conjugation |
18 |
| Integrated Assessment of the Clinical Performance of GalNAc(3)-Conjugated 2 '-O-Methoxyethyl Chimeric Antisense Oligonucleotides: I. Human Volunteer Experience |
15 |
| The Effects of 2-O-Methoxyethyl Oligonucleotides on Renal Function in Humans |
13 |
| Evaluation and Reduction of CRISPR Off-Target Cleavage Events |
13 |
| Guidelines for Experiments Using Antisense Oligonucleotides and Double-Stranded RNAs |
11 |
| Receptor-Mediated Uptake of Phosphorothioate Antisense Oligonucleotides in Different Cell Types of the Liver |
11 |
| Cell-Penetrating Peptide Conjugates of Steric Blocking Oligonucleotides as Therapeutics for Neuromuscular Diseases from a Historical Perspective to Current Prospects of Treatment |
10 |
| MicroRNAs Enable mRNA Therapeutics to Selectively Program Cancer Cells to Self-Destruct |
9 |
| A Sequel to the Eteplirsen Saga: Eteplirsen Is Approved in the United States but Was Not Approved in Europe |
8 |
| Lipid Conjugates Enhance Endosomal Release of Antisense Oligonucleotides Into Cells |
8 |
| In Vivo Silencing of MicroRNA-132 Reduces Blood Glucose and Improves Insulin Secretion |
7 |
| Evaluating the Impact of Variable Phosphorothioate Content in Tricyclo-DNA Antisense Oligonucleotides in a Duchenne Muscular Dystrophy Mouse Model |
6 |
| Endosomal Escape of ASOs Internalized by Stabilin Receptors Is Regulated by Rab5C and EEA1 During Endosomal Maturation |
6 |
| Current Status of Messenger RNA Delivery Systems |
6 |
| Aptamer-Mediated Delivery and Cell-Targeting Aptamers: Room for Improvement |
5 |
| Activation of Frataxin Protein Expression by Antisense Oligonucleotides Targeting the Mutant Expanded Repeat |
5 |
| Dose-Dependent Lowering of Mutant Huntingtin Using Antisense Oligonucleotides in Huntington Disease Patients |
5 |
| The Pharmacokinetics of 2 '-O-Methyl Phosphorothioate Antisense Oligonucleotides: Experiences from Developing Exon Skipping Therapies for Duchenne Muscular Dystrophy |
4 |
| DNA Aptamers for the Malignant Transformation Marker CD24 |
4 |
| Targeting Translation Termination Machinery with Antisense Oligonucleotides for Diseases Caused by Nonsense Mutations |
4 |
| IONIS-PKKRx a Novel Antisense Inhibitor of Prekallikrein and Bradykinin Production |
4 |
| RNA Interference-Based Cancer Drugs: The Roadblocks, and the Delivery of the Promise |
4 |
| Quantification of siRNA-Antibody Conjugates in Biological Matrices by Triplex-Forming Oligonucleotide ELISA |
4 |
| Formulation of Biocompatible Targeted ECO/siRNA Nanoparticles with Long-Term Stability for Clinical Translation of RNAi |
3 |
| Effect of Sugar 2 ',4 '-Modifications on Gene Silencing Activity of Small Interfering RNA Duplexes |
3 |
| Inhibition of Human Papillomavirus Type 16 Infection Using an RNA Aptamer |
3 |
| Phosphorothioate Antisense Oligonucleotides Bind P-Body Proteins and Mediate P-Body Assembly |
3 |
| Optimization of a 40-mer Antimyelin DNA Aptamer Identifies a 20-mer with Enhanced Properties for Potential Multiple Sclerosis Therapy |
3 |
| Structural Studies and Gene Silencing Activity of siRNAs Containing Cationic Phosphoramidate Linkages |
3 |
| Duplex RNAs and ss-siRNAs Block RNA Foci Associated with Fuchs' Endothelial Corneal Dystrophy |
3 |
| Chronic Toxicity Assessment of 2 '-O-Methoxyethyl Antisense Oligonucleotides in Mice |
3 |
| Cell-SELEX-Based Identification of a Human and Mouse Cross-Reactive Endothelial Cell-Internalizing Aptamer |
3 |
| Cholesterol-GalNAc Dual Conjugation Strategy for Reducing Renal Distribution of Antisense Oligonucleotides |
2 |
| The Munich Meeting: Medical Maturation, More Mechanisms, and Milasen |
2 |
| Development of a Nanostructured RNA/DNA Assembly as an Adjuvant Targeting Toll-Like Receptor 7/8 |
2 |
| Double-Stranded DNA Fragments Bearing Unrepairable Lesions and Their Internalization into Mouse Krebs-2 Carcinoma Cells |
2 |
| Use of C9/11 Mismatch Control Small Interfering RNA Reveals Sequence-Related Off-Target Effect on Coagulation of an Small Interfering RNA Targeting Mouse Coagulation Factor XII |
2 |
| Nucleic Acid Therapies for Cystic Fibrosis |
2 |
| Intracerebroventricular Administration of a 2 '-O-Methyl Phosphorothioate Antisense Oligonucleotide Results in Activation of the Innate Immune System in Mouse Brain |
2 |
| Keratinocyte Growth Factor Modified Messenger RNA Accelerating Cell Proliferation and Migration of Keratinocytes |
2 |
| Lowering Mutant Huntingtin Using Tricyclo-DNA Antisense Oligonucleotides As a Therapeutic Approach for Huntington's Disease |
2 |
| G-Quadruplex Structure Improves the Immunostimulatory Effects of CpG Oligonucleotides |
2 |
| Nonclinical Exon Skipping Studies with 2 '-O-Methyl Phosphorothioate Antisense Oligonucleotides in mdx and mdx-utrn-/- Mice Inspired by Clinical Trial Results |
2 |
| Rationally Designed Small Nucleic Acid-Based Inhibitors of CRISPR-Cas9 |
2 |
| Antitumor Response by Endoplasmic Reticulum-Targeting DNA Vaccine Is Improved by Adding a KDEL Retention Signal |
1 |
