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SEQUENCE OF SYNTHETIC MECP2 FOR PROTEIN REPLACEMENT THERAPY.

机译:用于蛋白质替换治疗的合成MECP2的序列。

摘要

A polypeptide comprising (i) a MeCP2 protein or biologically active fragment or a derivative of said protein or fragment, and (ii) a protein transduction domain, wherein the MeCP2 protein is fused with the protein transduction domain , to use in a method to treat a neurodegenerative disease or a neurodevelopmental disease; wherein said fragment comprises at least 150 amino acids of the MeCP2 protein; and wherein said derivative of said protein or fragment has at least 60% identity with said MeCP2 protein or fragment thereof; and wherein said fragment or said derivative has at least 60% of said biological activity, as determined by an in vitro transcription assay described by Nan, X et al., Cell 88: 471-481 (1997).
机译:一种多肽,其包含(i)MeCP2蛋白或生物活性片段或所述蛋白或片段的衍生物,和(ii)蛋白转导结构域,其中MeCP2蛋白与蛋白转导结构域融合,用于治疗方法神经退行性疾病或神经发育疾病;其中所述片段包含MeCP2蛋白的至少150个氨基酸;其中所述蛋白质或片段的所述衍生物与所述MeCP2蛋白质或其片段具有至少60%的同一性;其中所述片段或所述衍生物具有至少60%的所述生物活性,如通过Nan,X等人,Cell 88:471-481(1997)所述的体外转录测定法所测定的。

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