Chapter title |
Exon Skipping by Ultrasound-Enhanced Delivery of Morpholino with Bubble Liposomes for Myotonic Dystrophy Model Mice
|
---|---|
Chapter number | 30 |
Book title |
Exon Skipping and Inclusion Therapies
|
Published in |
Methods in molecular biology, January 2018
|
DOI | 10.1007/978-1-4939-8651-4_30 |
Pubmed ID | |
Book ISBNs |
978-1-4939-8650-7, 978-1-4939-8651-4
|
Authors |
Negishi, Yoichi, Endo-Takahashi, Yoko, Ishiura, Shoichi, Yoichi Negishi, Yoko Endo-Takahashi, Shoichi Ishiura |
Abstract |
Abnormal splicing of the chloride channel 1 (CLCN1) gene causes myotonic dystrophy type 1 (DM1). Therefore, controlling the alternative splicing process of this gene by antisense oligonucleotides can be a promising treatment for DM1. In this study, we describe an efficient phosphorodiamidate morpholino oligomer (PMO) delivery method by ultrasound-mediated bubble liposomes, which is a known gene delivery tool with ultrasound exposure, to treat skeletal muscles in a DM1 mouse model, HSALR. Effective delivery of PMO using this technique can help control the alternative splicing of the Clcn1 gene via exon skipping and enhance the expression of Clcn1 protein in skeletal muscles and the amelioration of myotonia. Thus, exon skipping by PMO delivery with ultrasound-mediated BLs may be feasible in myotonic dystrophy model mice. |
X Demographics
Geographical breakdown
Country | Count | As % |
---|---|---|
United States | 1 | 50% |
Unknown | 1 | 50% |
Demographic breakdown
Type | Count | As % |
---|---|---|
Members of the public | 1 | 50% |
Scientists | 1 | 50% |
Mendeley readers
Geographical breakdown
Country | Count | As % |
---|---|---|
Unknown | 13 | 100% |
Demographic breakdown
Readers by professional status | Count | As % |
---|---|---|
Student > Bachelor | 3 | 23% |
Researcher | 2 | 15% |
Student > Ph. D. Student | 1 | 8% |
Other | 1 | 8% |
Unknown | 6 | 46% |
Readers by discipline | Count | As % |
---|---|---|
Biochemistry, Genetics and Molecular Biology | 2 | 15% |
Business, Management and Accounting | 1 | 8% |
Agricultural and Biological Sciences | 1 | 8% |
Chemistry | 1 | 8% |
Unknown | 8 | 62% |