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V2 Pharmacoperone
This invention reports pharmacoperones for their novel use of therapeutics in the treatment of nephrogenic diabetes insipidus (NDI). Pharmacoperones are small molecules that enter the cell and serve as a scaffolding to allow mutated proteins to route correctly. In NDI, there is a mutation in the V2 receptor protein. Here, the inventor has reported over...
Published: 7/2/2018
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Inventor(s):
Michael Conn
Keywords(s):
Category(s):
Cancer Diagnostics
Development of a Phenotypic High-Content Assay to Identify Pharmacoperone Drugs for the Treatment of Primary Hyperoxaluria Type 1 by High-Throughput Screening
Hyperoxalosis is a deadly disease for which the best current therapy is dialysis or organ transplant. The underlying cause of some forms of the disease is the misrouting of a specific enzyme; alanine:glyoxylate aminotransferase (AGT) to the mitochondira inststead of the peroxisome. Pharmacoperones are small molecules that can restore proper function...
Published: 7/2/2018
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Inventor(s):
Michael Conn
Keywords(s):
Category(s):
Pharmaceutical
,
Assay