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Efficient systemic treatment of dystrophic conditions
Efficient systemic treatment of dystrophic conditions
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机译:营养不良状况的有效全身治疗
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摘要
A composition comprising a gene therapy product for use in the treatment of a dystrophic disease in a subject, advantageously in humans, wherein: - the gene therapy product comprises a nucleic acid sequence encoding a functional microdystrophin; - the composition is systemically administered.
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