Unknown

Dataset Information

0

Ultrasound-enhanced delivery of morpholino with Bubble liposomes ameliorates the myotonia of myotonic dystrophy model mice.


ABSTRACT: Phosphorodiamidate morpholino oligonucleotide (PMO)-mediated control of the alternative splicing of the chloride channel 1 (CLCN1) gene is a promising treatment for myotonic dystrophy type 1 (DM1) because the abnormal splicing of this gene causes myotonia in patients with DM1. In this study, we optimised a PMO sequence to correct Clcn1 alternative splicing and successfully remedied the myotonic phenotype of a DM1 mouse model, the HSALR mouse. To enhance the efficiency of delivery of PMO into HSALR mouse muscles, Bubble liposomes, which have been used as a gene delivery tool, were applied with ultrasound exposure. Effective delivery of PMO led to increased expression of Clcn1 protein in skeletal muscle and the amelioration of myotonia. Thus, PMO-mediated control of the alternative splicing of the Clcn1 gene must be important target of antisense therapy of DM1.

PROVIDER: S-EPMC3718203 | BioStudies |

REPOSITORIES: biostudies

Similar Datasets

| S-EPMC6018771 | BioStudies
| S-EPMC5272183 | BioStudies
2013-01-01 | S-EPMC3701899 | BioStudies
| S-EPMC6819114 | BioStudies
| S-EPMC6911202 | BioStudies
| S-EPMC7702137 | BioStudies
| E-GEOD-14691 | BioStudies
| S-EPMC2874217 | BioStudies
| S-EPMC7452101 | BioStudies
| S-EPMC6343479 | BioStudies