| abstract |
Provided herein are variant phenylalanine hydroxylase (PAH) polypeptides that are more stable and have higher activity than natural human PAH. Methods are also provided to treat phenylketonuria (PKU) and / or to reduce phenylalanine levels in an individual in need of it. In addition, provided herein are expression cassettes, vectors (eg, rAAV vectors), viral particles, pharmaceutical compositions, and kits for expressing the variant PAH polypeptide in an individual in need thereof. |