UMIN ID: UMIN000001996
Registered date:01/06/2009
Importance of checking exon skipping events prior to clinical trials using systemically delivered antisense morpholino in Duchenne muscular dystrophy patients
Basic Information
| Recruitment status | |
|---|---|
| Health condition(s) or Problem(s) studied | Duchenne muscular dystrophy |
| Date of first enrollment | 2009/06/01 |
| Target sample size | 10 |
| Countries of recruitment | Japan |
| Study type | Interventional |
| Intervention(s) | antisense morpholino |
Outcome(s)
| Primary Outcome | Fibroblasts from DMD patients were isolated, induced to differentiate to the myogenic lineage by AdMyoD (adenoviral vectors encoding MyoD regulated by CAG Promoter) and transfected with antisense morpholinos, which were designed to induce exon 51 skipping. The exon-skipping event was analyzed by RT-PCR. |
|---|---|
| Secondary Outcome |
Key inclusion & exclusion criteria
| Age minimum | Not applicable |
|---|---|
| Age maximum | 30years-old |
| Gender | Male |
| Include criteria | |
| Exclude criteria | The patients who can not consent the protocol. |
Related Information
| Primary Sponsor | Kumamoto University |
|---|---|
| Secondary Sponsor | |
| Source(s) of Monetary Support | This study was supported by the research grant (19-7) for Nervous and Mental Disorders from the Ministry of Health, Labour |
| Secondary ID(s) |
Contact
| public contact | |
| Name | |
| Address | Japan |
| Telephone | |
| kimusige@kumamoto-u.ac.jp | |
| Affiliation | Kumamoto University Graduate School Department of Child Development |
| scientific contact | |
| Name | Shigemi Kimura |
| Address | 1-1-1 Honjou Kumamoto 860-0811, Japan Japan |
| Telephone | |
| Affiliation | Kumamoto University Graduate School Department of Child Development, |