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GENE THERAPY COMPOSITION AND TREATMENT FOR MYH7-LINKED CARDIOMYOPATHY
GENE THERAPY COMPOSITION AND TREATMENT FOR MYH7-LINKED CARDIOMYOPATHY
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机译:基因治疗组合物和治疗MyH7联系心肌病
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摘要
Disclosed are a composition and method of treating or preventing cardiomyopathy in a human subject. In one embodiment, a method comprises delivering a gene therapy drug to cardiac tissue of the human subject. The gene therapy drug comprises: a first vector comprising a first portion of a polynucleotide sequence encoding for a therapeutic protein; and a second vector comprising a second portion of the polynucleotide sequence encoding for the therapeutic protein.
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