Title: Methods of treating Parkinson's disease using viral vectors

Abstract

Methods of delivering viral vectors, particularly recombinant adeno-associated virus (rAAV) virions, to the central nervous system (CNS) using convection enhanced delivery (CED) are provided. The rAAV virions include a nucleic acid sequence encoding a therapeutic polypeptide. The methods can be used for treating CNS disorders such as for treating Parkinson's Disease.

Inventors:

Bankiewicz, Krystof; Cunningham, Janet

Issue Date:

2016-11-15

Research Org.:

Lawrence Berkeley National Lab. (LBNL), Berkeley, CA (United States)

Sponsoring Org.:

USDOE

OSTI Identifier:

1332285

Patent Number(s):

9492415

Application Number:

13/647,155

Assignee:

The Regents of the University of California (Oakland, CA)

Patent Classifications (CPCs):

C - CHEMISTRY C12 - BIOCHEMISTRY C12N - MICROORGANISMS OR ENZYMES

A - HUMAN NECESSITIES A61 - MEDICAL OR VETERINARY SCIENCE A61K - PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES

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C - CHEMISTRY C12 - BIOCHEMISTRY C12N - MICROORGANISMS OR ENZYMES

C12N2750/14142 - virus or viral particle as vehicle, e.g. encapsulating small organic molecule

A - HUMAN NECESSITIES A61 - MEDICAL OR VETERINARY SCIENCE A61K - PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES

A61K38/45 - Transferases
A61K9/5184 - {Virus capsids or envelopes enclosing drugs

C - CHEMISTRY C12 - BIOCHEMISTRY C12Y - ENZYMES

C12Y401/01028 - Aromatic-L-amino-acid decarboxylase

A - HUMAN NECESSITIES A61 - MEDICAL OR VETERINARY SCIENCE A61K - PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES

A61K31/198 - Alpha-aminoacids, e.g. alanine, edetic acids [EDTA],

A - HUMAN NECESSITIES A01 - AGRICULTURE A01N - PRESERVATION OF BODIES OF HUMANS OR ANIMALS OR PLANTS OR PARTS THEREOF

A01N63/00 - Biocides, pest repellants or attractants, or plant growth regulators containing microorganisms, viruses, microbial fungi, animals or substances produced by, or obtained from microorganisms, viruses, microbial fungi or animals, e.g. enzymes or fermentates

A - HUMAN NECESSITIES A61 - MEDICAL OR VETERINARY SCIENCE A61P - SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS

A61P25/16 - Anti-Parkinson drugs

A - HUMAN NECESSITIES A61 - MEDICAL OR VETERINARY SCIENCE A61K - PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES

A61K48/0075 - {characterised by an aspect of the delivery route, e.g. oral, subcutaneous}
A61K47/6901 - {Conjugates being cells, cell fragments, viruses, ghosts, red blood cells or viral vectors}
A61K51/0402 - {carboxylic acid carriers, fatty acids
A61K51/00 - Preparations containing radioactive substances for use in therapy or testing in vivo
A61K45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
A61K48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases

C - CHEMISTRY C12 - BIOCHEMISTRY C12N - MICROORGANISMS OR ENZYMES

C12N2750/14143 - viral genome or elements thereof as genetic vector

A - HUMAN NECESSITIES A61 - MEDICAL OR VETERINARY SCIENCE A61K - PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES

A61K38/51 - Lyases

C - CHEMISTRY C12 - BIOCHEMISTRY C12N - MICROORGANISMS OR ENZYMES

C12N15/63 - Introduction of foreign genetic material using vectors
C12N7/00 - Viruses

A - HUMAN NECESSITIES A61 - MEDICAL OR VETERINARY SCIENCE A61K - PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES

A61K35/761 - Adenovirus

C - CHEMISTRY C12 - BIOCHEMISTRY C12N - MICROORGANISMS OR ENZYMES

C12N15/86 - Viral vectors

A - HUMAN NECESSITIES A61 - MEDICAL OR VETERINARY SCIENCE A61K - PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES

A61K48/0025 - {wherein the non-active part clearly interacts with the delivered nucleic acid}

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DOE Contract Number:  

AC03-76SF00098; AC02-05CH11231

Resource Type:

Patent

Resource Relation:

Patent File Date: 2012 Oct 08

Country of Publication:

United States

Language:

English

Subject:

60 APPLIED LIFE SCIENCES; 59 BASIC BIOLOGICAL SCIENCES