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Nedosiran

CAS No. : 2266591-83-5

MCE 国际站：Nedosiran

产品活性：Nedosiran (DCR-PHXC) 是一种靶向乳酸脱氢酶 (LDH) 的 RNA 干扰剂 (RNAi)。Nedosiran 可用于研究并发终末期肾病 (ESRD)的原发性高草酸尿症 (PH)。Nedosiran 是一种 GalNAc-dsRNA 偶联物。

研究领域：Epigenetics

作用靶点：Small Interfering RNA (siRNA)

In Vitro: Nedosiran (DCR-PHXC) is an investigational ribonucleic acid interference (RNAi) therapy.
Nedosiran is a synthetic, double-stranded RNA oligonucleotide (i.e., small interfering RNA [siRNA]) designed to target the mRNA encoding LDHA.

In Vivo: Nedosiran is a RNAi therapy for primary hyperoxaluria that selectively reduce hepatic expression of lactate dehydrogenase.
Nedosiran specially inhibits hepatic expression of lactate dehydrogenase (LDHA).
Nedosiran is a double-strand siRNA molecule that is conjugated with GalNAc, which takes advantage of the aforementioned unique asialoglycoprotein receptor (ASGPR) delivery system in the liver. Nedosiran is administered by monthly subcutaneous injection. Nedosiran decreases plasma oxalate in animal models.

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