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AAV GENE THERAPY FOR TREATING NEPHROTIC SYNDROME
AAV GENE THERAPY FOR TREATING NEPHROTIC SYNDROME
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机译:AAV基因疗法治疗肾病综合症
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摘要
The present invention provides an adeno-associated virus (AAV) vector gene therapy for use in treating a monogenic form of nephrotic syndrome, wherein the AAV vector comprises a NS-associated transgene and minimal nephrin promoter NPHS1 or podocin promoter NPHS2.
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