1. Antibody-drug Conjugate/ADC Related Apoptosis Cytoskeleton
  2. Transferrin Receptor Antibody-Oligonucleotide Conjugates (AOCs) Dystrophin
  3. Delpacibart zotadirsen

Delpacibart zotadirsen (del-zota;AOC 1044) 是一种靶向肌营养不良蛋白 (dystrophin) 基因 44 号外显子突变位点的选择性外显子跳跃诱导剂,是一种抗体-寡核苷酸偶联物 (AOC),由磷酰二胺吗啉代寡核苷酸 (PMO) Zotadirsen (HY-177972) 和 TFRC 抗体 Delpacibart (HY-P990051) 偶联合成。Delpacibart zotadirsen 通过诱导 dystrophin 基因 44 号外显子跳跃,恢复基因阅读框,产生缩短但具有功能的 dystrophin 蛋白,从而改善肌纤维完整性。Delpacibart zotadirsen 可显著且持久降低血中肌酸激酶、丙氨酸转氨酶、天冬氨酸转氨酶及肌红蛋白水平至接近正常,改善肌肉损伤标志物,且安全性良好。Delpacibart zotadirsen 适用于 44 号外显子跳跃突变的杜氏肌营养不良症 (DMD44) 研究。

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Delpacibart zotadirsen

Delpacibart zotadirsen Chemical Structure

CAS No. : 2929240-20-8

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1 mg ¥13760
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  • 生物活性

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  • 参考文献

生物活性

Delpacibart zotadirsen (Del-zota), an antibody oligonucleotide conjugate (AOC), consists of a monoclonal antibody (Delpacibart) (HY-P990051) that binds to the transferrin receptor 1 (TfR1) conjugated to a phosphorodiamidate morpholino conjugate (PMO), Delpacibart zotadirsen is designed to deliver phosphorodiamidate morpholino oligomers (PMOs) to skeletal muscle and heart tissue to specifically skip exon 44 of the dystrophin gene and enable production of near-full length dystrophin. Delpacibart zotadirsen is used for the study of myotonic dystrophy type 1 (DM1)[1].

体外研究
(In Vitro)

Delpacibart zotadirsen 主要应用于 44 号外显子跳跃突变的杜氏肌营养不良症 (DMD44) 研究,可覆盖 7-27 岁 ambulatory 和非 ambulatory 患者背景[1]

MCE has not independently confirmed the accuracy of these methods. They are for reference only.

CAS 号
运输条件

Room temperature in continental US; may vary elsewhere.

储存方式

Please store the product under the recommended conditions in the Certificate of Analysis.

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产品名称:
Delpacibart zotadirsen
目录号:
HY-177564
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