产品
编 号：F142568
分子式：C19H40N4O14S
分子量：580.6
分子式：C19H40N4O14S
分子量：580.6
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CAS No: 1375073-94-1
产品详情
生物活性：
Exaluren (ELX-02) sulfate is an investigational, advanced synthetic eukaryotic ribosome selective glycoside (ERSG). Exaluren sulfate is being developed as a therapy for genetic diseases caused by nonsense mutations.
体内研究：
Exaluren (ELX-02) sulfate (10 and 30 mg/kg; repeat subcutaneous administration; twice weekly, total of 8 doses) shows accumulation in tissues that is dose dependent without gender difference.In plasma Exaluren (ELX-02) sulfate is rapidly absorbed with a Tmax of 0.25 h after both single (a single subcutaneous injection at 10 mg/kg at dose volume of 5 mL/kg) and repeated administration (twice weekly with 10 mg/kg/dose for 21 days; total of 7 administrations). Exaluren (ELX-02) sulfate is rapidly eliminated from plasma in a biphasic manner with the terminal half-life (T1/2) of 0.5 h. In a CtnsY226X nonsense mutant mouse, subcutaneous Exaluren (ELX-02) sulfate accumulates in kidney tissue without overt renal toxicity and that Exaluren (ELX-02) sulfate (10 mg/kg X2/week for 3 weeks) reduces renal cystine accumulation in vivo.Animal Model:Twenty-nine CtnsY226X/Y226X mice 5-7 month old
Dosage:10 and 30 mg/kg
Administration:Subcutaneous injection, at dose volume of 5 mL/kg, twice weekly for a period of 28 days (total of 8 doses)
Result:Highest levels were measured in the kidney, followed by spleen and liver, with lower levels in other tissues (lung, heart, cochlea and brain).
体外研究：
Exaluren (ELX-02) sulfate (100-400 μg/mL) is not toxic, and permits read-through of nonsense mutations in human cells.
Exaluren (ELX-02) sulfate is an investigational, advanced synthetic eukaryotic ribosome selective glycoside (ERSG). Exaluren sulfate is being developed as a therapy for genetic diseases caused by nonsense mutations.
体内研究：
Exaluren (ELX-02) sulfate (10 and 30 mg/kg; repeat subcutaneous administration; twice weekly, total of 8 doses) shows accumulation in tissues that is dose dependent without gender difference.In plasma Exaluren (ELX-02) sulfate is rapidly absorbed with a Tmax of 0.25 h after both single (a single subcutaneous injection at 10 mg/kg at dose volume of 5 mL/kg) and repeated administration (twice weekly with 10 mg/kg/dose for 21 days; total of 7 administrations). Exaluren (ELX-02) sulfate is rapidly eliminated from plasma in a biphasic manner with the terminal half-life (T1/2) of 0.5 h. In a CtnsY226X nonsense mutant mouse, subcutaneous Exaluren (ELX-02) sulfate accumulates in kidney tissue without overt renal toxicity and that Exaluren (ELX-02) sulfate (10 mg/kg X2/week for 3 weeks) reduces renal cystine accumulation in vivo.Animal Model:Twenty-nine CtnsY226X/Y226X mice 5-7 month old
Dosage:10 and 30 mg/kg
Administration:Subcutaneous injection, at dose volume of 5 mL/kg, twice weekly for a period of 28 days (total of 8 doses)
Result:Highest levels were measured in the kidney, followed by spleen and liver, with lower levels in other tissues (lung, heart, cochlea and brain).
体外研究：
Exaluren (ELX-02) sulfate (100-400 μg/mL) is not toxic, and permits read-through of nonsense mutations in human cells.
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