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Product details:
- Edition number 2
- Publisher Springer US
- Date of Publication 11 September 2026
- ISBN 9781071654750
- Binding Hardback
- No. of pages527 pages
- Size 254x178 mm
- Language English
- Illustrations XIV, 527 p. 9 illus. 700
Categories
Long description:
This new edition reflects the progression of gapmer research from conceptual foundations to experimental implementation and analytical detection. Beginning with a section covering historical perspectives and the clinical context of antisense gapmers, the book continues with molecular design principles, experimental strategies for developing effective antisense oligonucleotides, methods to evaluate their efficacy and selectivity in cellular and animal models, and analytical approaches for detecting and quantifying antisense oligonucleotides in biological samples. Written for the highly successful Methods in Molecular Biology series, chapters include the kind of detailed implementation advice needed to ensure success in the lab.
Authoritative and up-to-date, Gapmers: Methods and Protocols, Second Edition serves as a valuable resource for researchers and clinicians aiming to accelerate the development of next-generation antisense therapeutics.
MoreTable of Contents:
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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.
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