Phenylketonuria (PKU) is a genetic disease that leads to an accumulation of the amino acid phenylalanine in the blood, resulting in stunted growth, seizures, and cognitive impairment. Some moderate forms of PKU respond to tetrahydrobiopterin (BH4) supplementation, which lowers the levels of phenylalanine in the blood, and are known as BH4-responsive PKU. Today, chemically synthesized BH4 is used to treat BH4-responsive PKU and is the only source of therapeutic BH4 in the US. Our goal is to engineer the biological manufacturing of BH4 from glucose using Saccharomyces cerevisiae as the microbial host, resulting in the isolation of sufficient microbially produced BH4 for complete chemical characterization and future in vivo testing.

High-level microbial production of tetrahydrobiopterin in the yeast <i>Saccharomyces cerevisiae</i>

248th National Meeting (2014)

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High-level microbial production of tetrahydrobiopterin in the yeast Saccharomyces cerevisiae