Nonsense mutation read-through function of indole 3 methanol

Abstract

The invention discloses a nonsense mutation read-through function of indole 3 methanol. Preliminary experimental results show that coumestrol can partially recover expression of luciferase containingnonsense mutation in a cell model, and can partially recover expression of ATM genes containing nonsense mutation in an extracellular protein truncation experiment, and the coumestrol is expected to serve as a lead compound, is developed into a new drug through structural modification and modification, treats various diseases caused by nonsense mutation, and has potential medical application.

Description

technical field [0001] The invention belongs to the field of biomedicine, and in particular relates to the use of nonsense mutation read-through activity of indole 3 carbinol. Background technique [0002] Studies have found that 30% of human disease-causing gene mutations are nonsense mutations, which are related to the occurrence of many diseases including tumors and genetic diseases. Nonsense mutations can cause the codons encoding amino acids to be mutated into stop codons, and the appearance of premature stop codons will cause the termination of peptide chain synthesis, resulting in non-functional or incomplete truncated proteins; it will also cause mRNA to stay in the nucleus for a long time Prolonged, even through the nonsense-mediated mRNA decay (nonsense-mediated mRNA decay, NMD) pathway, the mRNA is selectively degraded, resulting in the loss of the encoded protein. [0003] Studies have shown that some compounds have nonsense mutation read-through activity. Under...

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Problems solved by technology

[0006] Aiming at the problems of the existing nonsense mutation read-through agents such as few types, high toxicity, and low read-through efficiency, the present invention proposes the use of indole 3 carbinol nonsense mutation read-through activity

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