CRISPR treatment shows promise in rare muscle disease
NCT ID NCT06594094
First seen Feb 17, 2026 · Last updated May 05, 2026 · Updated 11 times
Summary
This early-phase trial tested a CRISPR-based gene therapy (HG302) in 4 boys aged 4 to 8 with Duchenne muscular dystrophy (DMD), a severe muscle-wasting disease. The therapy aims to restore the missing dystrophin protein in muscle cells using a single intravenous injection. The main goal was to check safety, while also measuring how much dystrophin protein was produced and whether muscle function improved.
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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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Contacts and locations
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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
Conditions
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