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Importance of checking exon skipping events prior to clinical trials using systemically delivered antisense morpholino in Duchenne muscular dystrophy patients

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

Status
Unknown
Phases
Unknown
Study type
Interventional
Source
JPRN
Registry ID
JPRN-UMIN000001996
Enrollment
10
Registered
2009-06-01
Start date
2009-06-01
Completion date
Unknown
Last updated
2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Duchenne muscular dystrophy

Interventions

antisense morpholino

Sponsors

Kumamoto University
Lead Sponsor

Eligibility

Sex/Gender
Male

Inclusion criteria

Inclusion criteria: Duchenne muscular dystrophy, who is predicted of the restoration of the dystrophin open reading frame when the exon 51 was excluded.

Exclusion criteria

Exclusion criteria: The patients who can not consent the protocol.

Design outcomes

Primary

MeasureTime frame
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.—

Countries

Japan

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026