Splice switching : methods and protocols / edited by Yoshitsugu Aoki, Norio Motohashi.

Format:

Book

Contributor:

Aoki, Yoshitsugu, editor.

Motohashi, Norio, editor.

Series:

Methods in molecular biology (Clifton, N.J.) ; 1940-6029 v. 2963.

Methods in molecular biology, 1940-6029 ; 2963

Language:

English

Subjects (All):

RNA--Therapeutic use.

RNA.

RNA splicing--Laboratory manuals.

RNA splicing.

Gene therapy.

Genetic Therapy.

Medical Subjects:

Genetic Therapy.

Genre:

Laboratory manuals.

Physical Description:

1 online resource (xi, 226 pages) : illustrations (chiefly color).

Place of Publication:

New York, NY : Humana Press, [2026]

Summary:

This volume explores the latest advancements in the field of RNA therapeutics, with a particular focus on the development of exon skipping and inclusion therapies as targeted treatments for genetic diseases, as well as the evaluation methods used post-treatment. The chapters in this book cover both established methods and novel techniques used my researchers working on developing therapies that have proven to be promising for several diseases, including Duchenne muscular dystrophy (DMD), spinal muscular atrophy (SMA), and other genetic disorders characterized by abnormal splicing events. 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. Cutting-edge and thorough, Splice Switching: Methods and Protocols is a valuable resource for researchers across various fields. This book will help in the advancement of basic RNA biology research and aid in accelerating therapeutic developments. .

Contents:

DG9-conjugated morpholino-based exon 51-skipping therapy for Duchenne muscular dystrophy / Md Nur Ahad Shah, Laura Edellein Sutanto, and Toshifumi Yokota

DG9-conjugated morpholino rescues phenotype in spinal muscular atrophy mice / Umme Sabrina Haque, Melissa Kohut, and Toshifumi Yokota

Cardio-physiological evaluations in a dog model with Duchenne muscular dystrophy / Koichi Kimura and Hiroyuki Morita

Splice modulation studies using urine-derived cells / Katsuhiko Kunitake and Yoshitsugu Aoki

Lipidomic sample preparation to detect phosphatidylcholine alterations in skeletal muscle / William J. Valentine, Suzumi M. Tokuoka, Yoshihiro Kita, and Hideo Shindou

Evaluation for the efficacy of exon-skipping therapy using in vivo assessment of skeletal muscle function and fragility / Yuki Ashida, Katsura Minegishi, Norio Motohashi, Yoshitsugu Aoki, and Takashi Yamada

Characterization and phenotypic assessment of zebrafish disease models for splice modulation studies / Holly R. Thomas, Katherine G. English, Matthew S. Alexander, and John M. Parant

Preparation of size-defined PEG-grafted copolymers as a polymeric nanoruler for size optimization in passive targeting / Yukine Ishibashi, Mitsuru Naito, Chun Yin Jerry Lau, Yoshitsugu Aoki, and Kanjiro Miyata

Splice modulation studies using reporter mice / Katsura Minegishi, Tsukasa Tominari, and Yoshitsugu Aoki

Clinical variant databases and machine learning prediction supporting genomic medicine / Mayumi Kamada and Yosuke Kawai

In vivo evaluation of CTG repeat-affected muscle pathology in a myotonic dystrophy model mouse using electromyography and fluorescence in situ hybridization / Yusuke Echigoya, Katsura Minegishi, Masayuki Nakamori, and Yoshitsugu Aoki

Utilization of induced pluripotent stem cell-derived neurons to investigate the splice-modification efficacy of splice-switching drug candidates / Yukiko Ueta, Hiroaki Ohara, Tomonari Awaya, and Masatoshi Hagiwara

Development of electric field stimulation (EFS)-mediated skeletal muscle training with iPSCs / Tomoya Uchimura and Hidetoshi Sakurai

Neurological behavioral assessment of exon-skipping therapy for neuromuscular disorders using displaced object recognition test, open field test, and three-chamber social approach test / Hiroya Ono and Yoshitsugu Aoki

Analysis of prefrontal–amygdala synaptic functions using optogenetics and patch-clamp recording / Hiroshi Kuniishi and Eri Takeuchi

Digital outcome measures for gait and spontaneous locomotor activity in dystrophic dogs / Mutsuki Kuraoka, Karin Watanabe, Takahiro Kawashima, Yuko Nitahara-Kasahara, Hisateru Tachimori, Shin’ichi Takeda, Katsura Minegishi, and Yoshitsugu Aoki.

Notes:

Includes bibliographical references and index.

Online resource; title from PDF title page (SpringerLink, viewed October 17, 2025).

Other Format:

Print version: Splice switching

ISBN:

9781071647387

1071647385

OCLC:

1545571568

Publisher Number:

CIPO000296250

Access Restriction:

Restricted for use by site license