Gapmers

Invention and Early History.- Development and Clinical Applications of Antisense Oligonucleotide Gapmers.- Knocking Down Long Non-coding RNAs using Antisense Oligonucleotide Gapmers.- Development of Antisense Oligonucleotide Gapmers for the Treatment of Huntington’s Disease.- Development of Antisense Oligonucleotide Gapmers for the Treatment of Dyslipidemia and Lipodystrophy.- Inotersen for the Treatment of Hereditary Transthyretin Amyloidosis.- Degradation of Toxic RNA in Myotonic Dystrophy Using Gapmer Antisense Oligonucleotides.- DNA/RNA Heteroduplex Oligonucleotide for Highly Efficient Gene Silencing.- Tips for Successful lncRNA Knockdown using Gapmers.- Calcium-Mediated In Vitro Transfection Technique of Oligonucleotides with Broad Chemical Modification Compatibility.- Evaluating the Knockdown Activity of MALAT1 ENA Gapmers in vitro.- Albumin-binding Fatty Acid-modified Gapmer Antisense Oligonucleotides for Modulation of Pharmacokinetics.- The use of Gapmers for in vivo Suppression of Hepatic mRNA Targets.- Development of LNA Gapmer Oligonucleotide-Based Therapy for ALS/ FTD Caused by the C9orf72 Repeat Expansion.- Targeted Gene Silencing in Malignant Hematolymphoid Cells using GapmeR.- Gapmer Antisense Oligonucleotides to Selectively Suppress the Mutant allele in COL6A Genes in Sominant Ullrich Congenital Muscular Dystrophy.- Detection of Locked Nucleic Acid Gapmers from Mouse Samples using ELISA.