Experimental gene therapy targets rare form of duchenne muscular dystrophy
NCT ID NCT04240314
First seen Jun 26, 2026 · Last updated Jun 26, 2026
Summary
This early-stage trial tested a single-dose gene therapy called scAAV9.U7.ACCA in 3 boys with Duchenne muscular dystrophy caused by a duplication of exon 2. The therapy was given through a vein and aimed to help muscle cells produce dystrophin, a protein missing in this disease. The main goal was to check for serious side effects, with a secondary look at whether dystrophin levels increased in muscle biopsies.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
scAAV9.U7.ACCA (a gene therapy given as a single injection into a vein)
What this could lead to
If successful, this could point toward a treatment that helps boys with Duchenne muscular dystrophy produce a missing protein, potentially slowing muscle damage.
What could go wrong
This is a very early, small trial (only 3 participants) focused on safety, not effectiveness. The therapy may not work or could cause serious side effects.
Disclaimer
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the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States