Patent ID: 11903985
Assignee: PREVAIL THERAPEUTICS, INC.
Field: Pharmaceuticals (Chemistry)
Classification: CPC A  C | IPC A  C

Claim 22:
23. A method for treating a subject having Parkinson's disease with a GBA1 mutation, the method comprising administering to the subject a rAAV comprising:
(i) a rAAV vector comprising a nucleic acid comprising, in 5′ to 3′ order:
(a) an AAV2 ITR;
(b) a CMV enhancer;
(c) a CBA promoter;
(d) a transgene insert encoding a Gcase protein, wherein the transgene insert comprises the nucleotide sequence of SEQ ID NO: 15;
(e) a WPRE;
(f) a Bovine Growth Hormone polyA signal tail; and
(g) an AAV2 ITR; and

(ii) an AAV9 capsid protein;
wherein the rAAV is administered to the subject at a dose ranging from about 5×1013 vg to about 5×1014 vg.