The invention relates to the use of adeno-associated virus (AAV) vectors to achieve long term expression of a transgene in the liver of a juvenile subject. The invention includes the stable long-term amelioration of disease symptoms of the subjection following a single administration of an AAV vector to a juvenile subject, wherein the AAV vector delivers the transgene to the subject’s liver.本發明係關於腺相關病毒(AAV)載體在幼年受試者肝臟中達成轉殖基因長期表現的用途。本發明包括在將AAV載體單次投與幼年受試者後，受試者的疾病症狀得到長期穩定的改善，其中所述AAV載體將轉殖基因遞送至受試者的肝臟。
