Gapmers: Methods and Protocols

Front Matter ....Pages i-xi
Front Matter ....Pages 1-1
 Invention and Early History of Gapmers  (Kenji Rowel Q. Lim, Toshifumi Yokota)....Pages 3-19
 Development and Clinical Applications of Antisense Oligonucleotide Gapmers  (Leanna Chan, Toshifumi Yokota)....Pages 21-47
 Knocking Down Long Noncoding RNAs Using Antisense Oligonucleotide Gapmers  (Rika Maruyama, Toshifumi Yokota)....Pages 49-56
 Development of Antisense Oligonucleotide Gapmers for the Treatment of Huntington’s Disease  (Tejal Aslesh, Toshifumi Yokota)....Pages 57-67
 Development of Antisense Oligonucleotide Gapmers for the Treatment of Dyslipidemia and Lipodystrophy  (Tejal Aslesh, Toshifumi Yokota)....Pages 69-85
 Inotersen for the Treatment of Hereditary Transthyretin Amyloidosis  (Maria Mahfouz, Rika Maruyama, Toshifumi Yokota)....Pages 87-98
 Degradation of Toxic RNA in Myotonic Dystrophy Using Gapmer Antisense Oligonucleotides  (Quynh Nguyen, Toshifumi Yokota)....Pages 99-109
Front Matter ....Pages 111-111
 DNA–RNA Heteroduplex Oligonucleotide for Highly Efficient Gene Silencing  (Rintaro Iwata Hara, Kotaro Yoshioka, Takanori Yokota)....Pages 113-119
 Tips for Successful lncRNA Knockdown Using Gapmers  (Kim A. Lennox, Mark A. Behlke)....Pages 121-140
 Calcium-Mediated In Vitro Transfection Technique of Oligonucleotides with Broad Chemical Modification Compatibility  (Fumito Wada, Shin-ichiro Hori, Satoshi Obika, Tsuyoshi Yamamoto)....Pages 141-154
 Evaluating the Knockdown Activity of MALAT1 ENA Gapmers In Vitro  (Shinzo Iwashita, Takao Shoji, Makoto Koizumi)....Pages 155-161
 Albumin-Binding Fatty Acid–Modified Gapmer Antisense Oligonucleotides for Modulation of Pharmacokinetics  (Yunpeng Cai, Chenguang Lou, Jesper Wengel, Kenneth A. Howard)....Pages 163-174
Front Matter ....Pages 175-175
 The Use of Gapmers for In Vivo Suppression of Hepatic mRNA Targets  (David S. Greenberg, Yonat Tzur, Hermona Soreq)....Pages 177-184
 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 et al.)....Pages 185-208
 Targeted Gene Silencing in Malignant Hematolymphoid Cells Using GapmeR  (Atish Kizhakeyil, Mobashar Hussain Urf Turabe Fazil, Navin Kumar Verma)....Pages 209-219
 Gapmer Antisense Oligonucleotides to Selectively Suppress the Mutant Allele in COL6A Genes in Dominant Ullrich Congenital Muscular Dystrophy  (Sara Aguti, Elena Marrosu, Francesco Muntoni, Haiyan Zhou)....Pages 221-230
Front Matter ....Pages 231-231
 Detection of Locked Nucleic Acid Gapmers from Mouse Muscle Samples Using ELISA  (Kenji Rowel Q. Lim, Quynh Nguyen, Toshifumi Yokota)....Pages 233-239
Back Matter ....Pages 241-247