CRISPR therapy shows promise in early trial for fatal muscle disease
NCT ID NCT06594094
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-phase study tested a new gene editing treatment called HG302 in 4 boys with Duchenne muscular dystrophy (DMD), a severe muscle-wasting disease. The therapy uses CRISPR technology to fix the genetic error and help muscles produce dystrophin, a protein essential for muscle strength. The main goal was to check safety, but researchers also looked for signs that the treatment restored dystrophin and improved muscle function.
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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.
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Locations
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Shanghai Children s Medical Center Affiliated to Shanghai Jiao Tong University School of Medical
Shanghai, Shanghai Municipality, China