IP Library Granted Patent US 8,309,355
Granted Patent B2
US 8,309,355 · App. 12/384,935 · Granted Nov 13, 2012

Methods of treating Parkinson's disease using viral vectors

Assignees: Genzyme Corporation; The Regents of the University of California
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Quick Facts
Patent No.
US 8,309,355
App. No.
12/384,935
Granted
Nov 13, 2012
Kind
B2
Abstract

Methods of delivering viral vectors, particularly recombinant AAV virions, to the central nervous system (CNS) are provided for the treatment of CNS disorders, particularly those disorders which involve the neurotransmitter dopamine. The methods entail providing rAAV virions that comprise a transgene encoding aromatic amino acid decarboxylase (AADC) and administering the virions to the brain of a mammal using a non-manual pump.

Claims (14)

1. A method of increasing dopamine levels in a mammal, said method comprising:

(a) providing a composition comprising at least 2.5×10 9 recombinant adeno-associated virus (rAAV) virions, wherein said rAAV virions comprise a transgene encoding aromatic amino acid decarboxylase (AADC); and

(b) administering said composition directly to the brain of said mammal using a non-manual pump, to increase dopamine levels, wherein said composition is administered such that distribution of said rAAV virions is over an area greater than 5 mm 2 .

2. The method of claim 1 , wherein said pump is an infusion pump or an osmotic pump.

3. The method of claim 1 , wherein said composition is administered to striatum of the brain of said mammal.

4. The method of claim 1 , wherein said composition is administered under a pressure gradient.

5. The method of claim 1 , wherein said composition comprises at least 2.5×10 10 rAAV virions.

6. The method of claim 1 , wherein said mammal suffers from a central nervous system (CNS) disorder.

7. The method of claim 6 , wherein said CNS disorder is Parkinson's disease.

8. The method of claim 1 , further comprising administering L-dopa in said mammal.

9. The method of claim 8 , wherein said L-dopa is administered by systemic administration.

10. The method of claim 9 , wherein said systemic administration is oral.

11. The method of claim 8 , further comprising administering carbidopa.

12. The method of claim 1 , wherein at least 2.5×10 10 rAAV virions are administered such that distribution of said rAAV virions is over a volume of at least 250 mm 3 .

Assignments (1)
CONFIRMATORY LICENSE Recorded Apr 2, 2010
From: REGENTS OF THE UNIVERSITY OF CALIFORNIA, THE
To: UNITED STATES DEPARTMENT OF ENERGY
Reel/Frame 024178/0468 →
Continuity (6)
Division 11102521 · Apr 8, 2005
Continuation 09887854 · Jun 21, 2001
Continuation 09320171 · May 26, 1999
Provisional Application 60134748 · May 18, 1999
Provisional Application 60086949 · May 27, 1998
Related Publication 20100104537A1 · Apr 29, 2010