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

摘要

Methods for delivering a heterologous gene to a mammalian subject usingrecombinant adeno-associated virus (rAAV) virions are described. RecombinantAAV virions containing a heterologous gene encoding a metabolic protein aredelivered to a mammalian subject having a metabolic disorder. The rAAV virion-delivered heterologous gene is expressed at a therapeutic level therebyameliorating a sign or symptom of the metabolic disease. Exemplary examples ofmetabolic diseases are those caused by defects in aromatic amino acidmetabolism. Exemplary examples of heterologous genes include those encoding anaromatic amino acid hydroxylase, aromatic amino acid decarboxylase, andenzymes involved in tetrahydrobiopterin synthesis. Methods for treatingphenylketonuria are also described.