GJ-103 is an active analog of the read-through compound GJ072. GJ-103 showed activity comparable to stop codons (TGA, TAG, and TAA) PTC124 and RTC13. Early structure-activity relationship studies generated eight active analogs of GJ072.
|Body Surface Area (m2)||0.007||0.025||0.15||0.05||0.02||0.5|
|Animal A (mg/kg) = Animal B (mg/kg) multiplied by||Animal B Km|
|Animal A Km|
For example, to modify the dose of resveratrol used for a mouse (22.4 mg/kg) to a dose based on the BSA for a rat, multiply 22.4 mg/kg by the Km factor for a mouse and then divide by the Km factor for a rat. This calculation results in a rat equivalent dose for resveratrol of 11.2 mg/kg.
A new series of small molecular weight compounds induce read through of all three types of nonsense mutations in the ATM gene.
Du L, et al. Mol Ther. 2013 Sep;21(9):1653-60. PMID: 23774824.
Elamipretide, also known as MTP-131, is a cardiolipin peroxidase inhibitor and mitochondria-targeted peptide.
Hexacosanoic acid is a long-chain fatty acid related to various diseases such as adrenoleukodystrophy (ALD), adrenomyeloneuropathy (AMN) and atherosclerosis.
Corn oil, extracted from the germ of corn, can be used as a carrier for drug molecules and has anti-tumor activity.
Palosuran, also known as ACT-058362, is a Urotensin-II receptor antagonist with an IC50 of 3.6±0.2 nM.
Dithiothreitol (DTT) is the common name for a small-molecule redox reagent known as Cleland's reagent; as a reducing or "deprotecting" agent for thiolated DNA.
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