Gapmers: Methods in Molecular Biology, cartea 3065

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 Gapmers for the Treatment of Huntington’s Disease.- Development of Antisense Oligonucleotide Gapmers for the Treatment of Dyslipidemia and Lipodystrophy.- Olezarsen for the Treatment of Hypertriglyceridemia.- Inotersen for the Treatment of Hereditary Transthyretin Amyloidosis.- Eplontersen for the Treatment of Hereditary Transthyretin Amyloidosis.- Using Antisense Oligonucleotides for the Degradation of Toxic RNA in Myotonic Dystrophy Type 1.- Development of Antisense Oligonucleotide Gapmers for Amyotrophic Lateral Sclerosis.- DNA/RNA Heteroduplex Oligonucleotide for Highly Efficient Gene Silencing.- LncRNA Knockdown Using Gapmer Antisense Oligonucleotides.- Evaluating the Knockdown Activity of MALAT1 ENA Gapmers In Vitro.- Albumin-Binding Fatty Acid-Modified Gapmer Antisense Oligonucleotides for Modulation of Pharmacokinetics.- Screening and Evaluation Scheme for the Identification of Antisense Oligonucleotides with Antiviral Activity Against SARS-CoV-2.- Gapmer and Mixmer Antisense Oligonucleotides to Selectively Suppress the Mutant Allele in COL6A Genes in Dominant Ullrich Congenital Muscular Dystrophy.- Viral RNA-Targeting Locked Nucleic Acid Gapmers: Design and In Vitro Evaluation.- Tissue Pharmacokinetics of Antisense Oligonucleotides.- Detection of Locked Nucleic Acid and 2’-O-Methoxyethyl Gapmers from Mouse Samples Using ELISA.