Researchers discover new factor in preventing phenylketonuria, offering new treatment strategy
Researchers at The University of Texas MD Anderson Cancer Center have discovered a critical new factor in regulating phenylalanine metabolism and, therefore, in preventing the inherited metabolic disorder phenylketonuria (PKU). The research also suggests a possible avenue for new treatments that may be effective for certain patients with PKU. The study demonstrates that the long noncoding RNA (lncRNA) HULC directly regulates the metabolic enzyme phenylalanine hydroxylase (PAH). Loss of the lncRNA resulted in excess phenylalanine and symptoms consist with...











