Patent ID: 11865188
Assignee: NATIONAL TAIWAN UNIVERSITY
Field: Pharmaceuticals (Chemistry)
Classification: CPC A  C | IPC A  C

Claim 0:
1. A method of treating aromatic L-amino acid decarboxylase (AADC) deficiency in a pediatric subject, the method comprising:
(a) providing a pharmaceutical formulation comprising an rAAV2-hAADC vector comprising (i) a wild type AAV2 capsid, and (ii) a recombinant DNA dopa decarboxylase (DDC) gene insert comprising a nucleic acid sequence encoding hAADC; and
(b) delivering the pharmaceutical formulation to at least one target site in the brain of the subject in a dose of an amount of at least 1.8×1011 vg; wherein the pharmaceutical formulation further comprises empty AAV2 capsids at a percentage from about 50% cp/cp up to about 90% cp/cp, wherein the pharmaceutical formulation is delivered by stereotaxy.