Methods and compositions for treating phenylketonuria
AssigneeChildren's National Medical Center
InventorsMarshall Summar, Juan Cabrera-Luque, Gary Cunningham, Dione T. Kobayashi, James G. McArthur
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Abstract
The present invention provides compositions and methods of treating hyperphenylalaninemia (e.g., phenylketonuria) in a subject in need thereof comprising administering to the subject an effective amount of a phenylalanine dehydrogenase (PheDH) polypeptide. The present invention also provides pharmaceutical formulations comprising PheDH for lowering the phenylalanine concentration in the subject (e.g., in the intestines and/or blood).
Claims20
A pharmaceutical formulation comprising a phenylalanine dehydrogenase polypeptide, or a functional fragment thereof, and a pharmaceutically-acceptable carrier, wherein the phenylalanine dehydrogenase polypeptide, or the functional fragment thereof, comprises an amino acid sequence having at least 80% sequence identity to SEQ ID NO:
- 1, wherein the pharmaceutical formulation is in a solid or semi-solid dosage form, or contained within an artificial membrane.
A method of reducing the level of phenylalanine in a subject in need thereof, comprising administering to the subject an effective amount of a phenylalanine dehydrogenase polypeptide, or a functional fragment thereof, wherein the phenylalanine dehydrogenase polypeptide or functional fragment thereof has at least about 80% sequence identity to SEQ ID NO:
- 1, and wherein the phenylalanine dehydrogenase polypeptide or functional fragment thereof is in a pharmaceutical formulation that is in a solid or semi-solid form, or contained within an artificial membrane.
A pharmaceutical formulation comprising a phenylalanine dehydrogenase polypeptide, or a functional fragment thereof, and a pharmaceutically-acceptable carrier, wherein the phenylalanine dehydrogenase polypeptide, or the functional fragment thereof, comprises an amino acid sequence having at least 80% sequence identity to SEQ ID NO:
- 1, and wherein the pharmaceutical formulation is in form of a tablet, suppository, pill, capsule, microsphere, powder, suspension, cream, ointment, or lotion.
Dependencies are inferred from the claim text.