Unique ID issued by UMIN | UMIN000001996 |
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Receipt number | R000002433 |
Scientific Title | Importance of checking exon skipping events prior to clinical trials using systemically delivered antisense morpholino in Duchenne muscular dystrophy patients |
Date of disclosure of the study information | 2009/06/01 |
Last modified on | 2009/05/21 20:37:44 |
Importance of checking exon skipping events prior to clinical trials using systemically delivered antisense morpholino in Duchenne muscular dystrophy patients
Checking of exon skipping using human cell
Importance of checking exon skipping events prior to clinical trials using systemically delivered antisense morpholino in Duchenne muscular dystrophy patients
Checking of exon skipping using human cell
Japan |
Duchenne muscular dystrophy
Neurology | Pediatrics |
Others
YES
Promising results of the antisense morpholino therapy have recently been reported on a genetic therapy aimed at restoring the reading frame of the dystrophin pre-mRNA in cells from mdx mouse model. Prior to the initiation of clinical treatment, it is very important to confirm the correct exon skipping events with antisense, because the sequences are different between the human and animal models and a few Duchenne type has in frame deletion of dystrophin. Therefore, we need a system that can easily screen sequences of the antisense and identify patients who are eligible for the therapy.
Efficacy
Confirmatory
Explanatory
Not applicable
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.
Interventional
Single arm
Randomized
Open -but assessor(s) are blinded
No treatment
1
Treatment
Medicine | Gene |
antisense morpholino
0 | years-old | <= |
30 | years-old | >= |
Male
Duchenne muscular dystrophy, who is predicted of the restoration of the dystrophin open reading frame when the exon 51 was excluded.
The patients who can not consent the protocol.
10
1st name | |
Middle name | |
Last name | Shigemi Kimura |
Kumamoto University Graduate School
Department of Child Development,
1-1-1 Honjou Kumamoto 860-0811, Japan
1st name | |
Middle name | |
Last name |
Kumamoto University Graduate School
Department of Child Development
kimusige@kumamoto-u.ac.jp
Kumamoto University
This study was supported by the research grant (19-7) for Nervous and Mental Disorders from the Ministry of Health, Labour
NO
2009 | Year | 06 | Month | 01 | Day |
Unpublished
2009 | Year | 05 | Month | 12 | Day |
2009 | Year | 06 | Month | 01 | Day |
2013 | Year | 03 | Month | 01 | Day |
2009 | Year | 05 | Month | 21 | Day |
2009 | Year | 05 | Month | 21 | Day |
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000002433
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