Cell-Penetrating Peptide Conjugates of Steric Blocking Oligonucleotides as Therapeutics for Neuromuscular Diseases from a Historical Perspective to Current Prospects of Treatment
来源期刊:Nucleic Acid TherapeuticsDOI:10.1089/nat.2018.0747
Guidelines for Experiments Using Antisense Oligonucleotides and Double-Stranded RNAs
来源期刊:Nucleic Acid TherapeuticsDOI:10.1089/nat.2018.0772
Evaluation and Reduction of CRISPR Off-Target Cleavage Events
来源期刊:Nucleic Acid TherapeuticsDOI:10.1089/nat.2019.0790
A Sequel to the Eteplirsen Saga: Eteplirsen Is Approved in the United States but Was Not Approved in Europe.
来源期刊:Nucleic acid therapeuticsDOI:10.1089/nat.2018.0756
In Vivo Silencing of MicroRNA-132 Reduces Blood Glucose and Improves Insulin Secretion.
来源期刊:Nucleic acid therapeuticsDOI:10.1089/nat.2018.0763
Lipid Conjugates Enhance Endosomal Release of Antisense Oligonucleotides Into Cells.
来源期刊:Nucleic acid therapeuticsDOI:10.1089/nat.2019.0794
Evaluating the Impact of Variable Phosphorothioate Content in Tricyclo-DNA Antisense Oligonucleotides in a Duchenne Muscular Dystrophy Mouse Model.
来源期刊:Nucleic acid therapeuticsDOI:10.1089/nat.2018.0773
Nuclear and Cytoplasmatic Quantification of Unconjugated, Label-Free Locked Nucleic Acid Oligonucleotides
来源期刊:Nucleic Acid TherapeuticsDOI:10.1089/nat.2019.0810
Quantification of siRNA-Antibody Conjugates in Biological Matrices by Triplex-Forming Oligonucleotide ELISA.
来源期刊:Nucleic acid therapeuticsDOI:10.1089/nat.2018.0770
Targeting Translation Termination Machinery with Antisense Oligonucleotides for Diseases Caused by Nonsense Mutations
来源期刊:Nucleic Acid TherapeuticsDOI:10.1089/nat.2019.0779
Nonclinical Safety Profile of Revusiran, a 1st-Generation GalNAc-siRNA Conjugate for Treatment of Hereditary Transthyretin-Mediated Amyloidosis
来源期刊:Nucleic Acid TherapeuticsDOI:10.1089/nat.2019.0796
Effect of Sugar 2′,4′-Modifications on Gene Silencing Activity of siRNA Duplexes
来源期刊:Nucleic Acid TherapeuticsDOI:10.1089/nat.2019.0792
Using a State-of-the-Art Toolbox to Evaluate Molecular and Functional Readouts of Antisense Oligonucleotide-Induced Exon Skipping in mdx Mice
来源期刊:Nucleic Acid TherapeuticsDOI:10.1089/nat.2019.0824
Formulation of Biocompatible Targeted ECO/siRNA Nanoparticles with Long-Term Stability for Clinical Translation of RNAi.
来源期刊:Nucleic acid therapeuticsDOI:10.1089/nat.2019.0784
Double-Stranded DNA Fragments Bearing Unrepairable Lesions and Their Internalization into Mouse Krebs-2 Carcinoma Cells.
来源期刊:Nucleic acid therapeuticsDOI:10.1089/nat.2019.0786
RNA Reduction and Hepatotoxic Potential Caused by Non-Gapmer Antisense Oligonucleotides.
来源期刊:Nucleic acid therapeuticsDOI:10.1089/nat.2018.0741
Phosphorothioate Antisense Oligonucleotides Bind P-Body Proteins and Mediate P-Body Assembly.
来源期刊:Nucleic acid therapeuticsDOI:10.1089/nat.2019.0806
Activation of Innate Immune Responses by a CpG Oligonucleotide Sequence Composed Entirely of Threose Nucleic Acid.
来源期刊:Nucleic acid therapeuticsDOI:10.1089/nat.2018.0751
Ligand Conjugated Multimeric siRNAs Enable Enhanced Uptake and Multiplexed Gene Silencing
来源期刊:Nucleic Acid TherapeuticsDOI:10.1089/nat.2019.0782
Metabolic Benefits of MicroRNA-22 Inhibition.
来源期刊:Nucleic acid therapeuticsDOI:10.1089/nat.2019.0820
Antisense-Mediated Skipping of Dysferlin Exons in Control and Dysferlinopathy Patient-Derived Cells.
来源期刊:Nucleic acid therapeuticsDOI:10.1089/nat.2019.0788
Expression of TNRC6 (GW182) Proteins Is Not Necessary for Gene Silencing by Fully Complementary RNA Duplexes
来源期刊:Nucleic Acid TherapeuticsDOI:10.1089/nat.2019.0815
Systemic Alanine Glyoxylate Aminotransferase mRNA Improves Glyoxylate Metabolism in a Mouse Model of Primary Hyperoxaluria Type 1.
来源期刊:Nucleic acid therapeuticsDOI:10.1089/nat.2018.0740
Development of a Nanostructured RNA/DNA Assembly as an Adjuvant Targeting Toll-Like Receptor 7/8.
来源期刊:Nucleic acid therapeuticsDOI:10.1089/nat.2019.0787
Endocytosis Controls Small Interfering RNA Efficiency: Implications for Small Interfering RNA Delivery Vehicle Design and Cell-Specific Targeting.
来源期刊:Nucleic acid therapeuticsDOI:10.1089/nat.2019.0804
G-Quadruplex Structure Improves the Immunostimulatory Effects of Cytosine-Phosphate-Guanine Oligonucleotides.
来源期刊:Nucleic acid therapeuticsDOI:10.1089/nat.2018.0761
Extrapolation of Oligonucleotide Dose Levels Used in Nonclinical Toxicity Studies to Selection of Safe Starting Dose Levels in Human Clinical Trials.
来源期刊:Nucleic acid therapeuticsDOI:10.1089/nat.2019.0781