The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.

The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.

Date of Patent:
Jun. 28, 2022

Filed:

Jul. 10, 2014
Applicant:

Genzyme Corporation, Cambridge, MA (US);

Inventors:

James Dodge, Bridgewater, NJ (US);

Seng Cheng, Bridgewater, NJ (US);

Assignee:

GENZYME CORPORATION, Cambridge, MA (US);

Attorney:
Primary Examiner:
Int. Cl.
CPC ...
A61K 48/00 (2006.01); C12N 15/79 (2006.01); C07H 21/04 (2006.01); C12N 15/86 (2006.01); A61K 38/47 (2006.01);
U.S. Cl.
CPC ...
A61K 48/0075 (2013.01); A61K 38/47 (2013.01); C12N 15/86 (2013.01); C12N 2750/14143 (2013.01); C12Y 302/01045 (2013.01);
Abstract

This disclosure provides methods and compositions for treating lysosomal storage diseases in a subject. In one aspect of the invention, a transgene product is delivered to a subject by administering a recombinant neurotrophic viral vector containing the transgene to the brain. The viral vector delivers the transgene to a region of the brain which is susceptible to infection by the virus and which expresses the encoded recombinant viral gene product. Also provided are compositions for delivery of a transgene product to a subject by administering a recombinant neurotrophic viral vector containing the transgene to the subject's brain. The transgene product may be any that is deficient in a lysosomal storage disease.


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