Correction of human nonsense mutation via adenine base editing for Duchenne muscular dystrophy treatment in mouse
Duchenne muscular dystrophy (DMD) is the most prevalent herediatry disease in men, characterized by dystrophin deficiency, progressive muscle wasting, cardiac insufficiency, and premature mortality, with no effective therapeutic options. Here, we investigated whether adenine base editing can correct...
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Main Authors: | , , , , , , , , , , , , , , , , |
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Format: | Book |
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Elsevier,
2024-06-01T00:00:00Z.
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A1234.567 |
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