| 生物活性 |
Delpacibart (del-zota, AOC 1044) is a selective exon skipping inducer targeting the exon 44 mutation site of the dystrophin gene, and is an antibody-oligonucleotide conjugate (AOC). Delpacibart induces exon 44 skipping of the dystrophin gene, restoring the gene reading frame and producing a shortened but functional dystrophin protein, thereby improving muscle fiber integrity. Delpacibart can significantly and persistently reduce serum levels of creatine kinase, alanine aminotransferase, aspartate aminotransferase, and myoglobin to near-normal levels, improving muscle damage markers, and has a good safety profile. Delpacibart is used in the study of Duchenne muscular dystrophy (DMD44) suitable for exon 44 skipping mutations.
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| 分子量 |
144.44 kDa
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| CAS号 |
2653183-66-3
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| 中文名称 |
Delpacibart
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| 运输条件 |
Shipping with dry ice.
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| 储存方式 |
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纯度： 98%
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