Article
Development of a phenotypic high-content assay to identify pharmacoperone drugs for the treatment of primary hyperoxaluria type 1 by high-throughput screening.
Assay and drug development technologies - 1 Jan 2000
Madoux Franck, Janovick Jo Ann, Smithson David, Fargue Sonia, Danpure Christopher J, Scampavia Louis, Chen Yih-Tai, Spicer Timothy P, Conn P Michael
Abstract excerpt
Primary hyperoxaluria is a severe disease for which the best current therapy is dialysis or organ transplantation. These are risky, inconvenient, and costly procedures. In some patients, pyridoxine treatment can delay the need for these surgical procedures. The underlying cause of particular forms of this disease is the misrouting of a specific enzyme, alanine:glyoxylate aminotransferase (AGT), to the...
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