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Purine derivative for use in the treatment or prevention of diseases caused by a nonsense mutation

机译:嘌呤衍生物,用于治疗或预防由无意义突变引起的疾病

摘要

The present disclosure relates to 2,6-diaminopurine (DAP) for use in the treatment of a disease caused by a nonsense mutation in a gene, leading to the premature insertion of a UGA stop codon.
机译:本公开涉及2,6-二氨基嘌呤(DAP),其用于治疗由基因的无义突变引起的疾病,该疾病导致UGA终止密码子的过早插入。

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