COMPOSITIONS FOR DELIVERING ENYZMES INVOLVED IN AMINO ACID METABOLISM USING RECOMBINANT ADEMO-ASSOCIATED VIRUS VIRONS (RAAV VIRONS), AND METHOD AND USE FOR TREATING AMINOI ACID METABOLIC DISORDERS USING SUCH RAAV VIRONS

摘要

Methods for delivering a heterologous gene to a mammalian subject using recombinant adeno-associated virus (rAAV) virions are described. Recombinant AAV virions containing a heterologous gene encoding a metabolic protein are delivered to a mammalian subject having a metabolic disorder. The rAAV virion-delivered heterologous gene is expressed at a therapeutic level thereby ameliorating a sign or symptom of the metabolic disease. Exemplary examples of metabolic diseases are those caused by defects in aromatic amino acid metabolism. Exemplary examples of heterologous genes include those encoding an aromatic amino acid hydroxylase, aromatic amino acid decarboxylase, and enzymes involved in tetrahydrobiopterin synthesis. Methods for treating phenylketonuria are also described.