| abstract |
The present invention relates to compositions and methods for treating L-amino aromatic aromatic decarboxylase (AADC) deficiency. The present invention relates to a method of treating AADC deficiency in a pediatric subject, comprising the steps of: (a) using a pharmaceutical composition comprising an rAAV2-hAADC vector, (b) stereotaxically administering a dose, at least about 1.8 x 1011 μg of the pharmaceutical composition to at least one target site in the brain of the subject; administering the pharmaceutical composition to the brain optionally by frameless stereotaxis, and the dose optionally being at least about 2.4 x 1011 μg and in certain embodiments, the pharmaceutical composition comprising a vector concentration. rAAV2-hAADC equal to about 5.7 x 1011 vg / mL. The present invention also relates to methods for treating L-amino aromatic aromatic decarboxylase (AADC) deficiency, the method optionally further comprising the step of administering a therapeutically effective dose of a dopamine antagonist, such as risperidone, about. The present invention also relates to methods for treating a deficiency of aromatic L-amino acid decarboxylase (AADC), the method optionally comprising the use of a pharmaceutical composition comprising a rAAV2-hAADC vector, and empty capsids. |