MORE ABOUT THIS BOOK
Main description:
This volume presents a comprehensive collection of detailed state-of-the-art protocols for gapmer-mediated RNA knockdown from leaders in the field. Chapters detail a historical and contemporary perspective of RNase H-inducing antisense oligonucleotides called gapmers and gapmer-mediated RNA knockdown for basic research and therapies. Written in the highly successful Methods in Molecular Biology series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and tips on troubleshooting and avoiding known pitfalls.
Authoritative and cutting-edge, Gapmers: Methods and Protocols aims to provide diverse applications of gapmers along with protocols that will assist readers in moving the frontier.
Contents:
Part I: Basics and Introduction
1. Invention and Early History
Kenji Rowel Q. Lim and Toshifumi Yokota
2. Development and Clinical Applications of Antisense Oligonucleotide Gapmers
Leanna Chan and Toshifumi Yokota
3. Knocking Down Long Non-coding RNAs using Antisense Oligonucleotide Gapmers
Rika Maruyama and Toshifumi Yokota
4. Development of Antisense Oligonucleotide Gapmers for the Treatment of Huntington's Disease
Tejal Aslesh and Toshifumi Yokota
5. Development of Antisense Oligonucleotide Gapmers for the Treatment of Dyslipidemia and Lipodystrophy
Tejal Aslesh and Toshifumi Yokota
6. Inotersen for the Treatment of Hereditary Transthyretin Amyloidosis
Maria Mahfouz, Rika Maruyama, and Toshifumi Yokota
7. Degradation of Toxic RNA in Myotonic Dystrophy Using Gapmer Antisense Oligonucleotides
Quynh Nguyen and Toshifumi Yokota
Part II: Design of Gapmers and Strategy
8. DNA/RNA Heteroduplex Oligonucleotide for Highly Efficient Gene Silencing
Rintaro Iwata Hara, Kotaro Yoshioka, and Takanori Yokota
9. Tips for Successful lncRNA Knockdown using Gapmers
Kim A Lennox and Mark A Behlke
10. Calcium-Mediated In Vitro Transfection Technique of Oligonucleotides with Broad Chemical Modification Compatibility
Fumito Wada, Shin-ichiro Hori, Satoshi Obika, and Tsuyoshi Yamamoto
11. Evaluating the Knockdown Activity of MALAT1 ENA Gapmers in vitro
Shinzo Iwashita, Takao Shoji, and Makoto Koizumi
12. Albumin-binding Fatty Acid-modified Gapmer Antisense Oligonucleotides for Modulation of Pharmacokinetics
Yunpeng Cai, Chenguang Lou, Jesper Wengel, and Kenneth A. Howard
Part III: In Vitro/In Vivo Evaluation of Gapmers and Antisense Oligonucleotides
13. The use of Gapmers for in vivo Suppression of Hepatic mRNA Targets
David S. Greenberg, Yonat Tzur, and Hermona Soreq
14. Development of LNA Gapmer Oligonucleotide-Based Therapy for ALS/ FTD Caused by the C9orf72 Repeat Expansion
Chaitra Sathyaprakash, Raquel Manzano, Miguel A. Varela, Yasumasa Hashimoto, Matthew J. A. Wood, Kevin Talbot, and Yoshitsugu Aoki
15. Targeted Gene Silencing in Malignant Hematolymphoid Cells using GapmeR
Atish Kizhakeyil, Mobashar Hussain Urf Turabe Fazil, and Navin Kumar Verma
16. Gapmer Antisense Oligonucleotides to Selectively Suppress the Mutant allele in COL6A Genes in Sominant Ullrich Congenital Muscular Dystrophy
Sara Aguti, Elena Marrosu, Francesco Muntoni, and Haiyan Zhou
Part IV: Detection of Gapmers
17. Detection of Locked Nucleic Acid Gapmers from Mouse Samples using ELISA
Kenji Rowel Q. Lim, Quynh Nguyen, and Toshifumi Yokota
PRODUCT DETAILS
Publisher: Springer (Springer-Verlag New York Inc.)
Publication date: September, 2020
Pages: 230
Weight: 693g
Availability: Available
Subcategories: Genetics