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:
Aug. 16, 2016

Filed:

Mar. 14, 2013
Applicants:

Brian K. Kaspar, Westerville, OH (US);

Kevin Foust, Westerville, OH (US);

Inventors:

Brian K. Kaspar, Westerville, OH (US);

Kevin Foust, Westerville, OH (US);

Assignee:

NATIONWIDE CHILDREN'S HOSPITAL, Columbus, OH (US);

Attorney:
Primary Examiner:
Assistant Examiner:
Int. Cl.
CPC ...
A61K 48/00 (2006.01); C12N 15/86 (2006.01); C07K 14/47 (2006.01); C12N 7/00 (2006.01);
U.S. Cl.
CPC ...
A61K 48/0075 (2013.01); A61K 48/005 (2013.01); C07K 14/47 (2013.01); C07K 14/4702 (2013.01); C12N 7/00 (2013.01); C12N 15/86 (2013.01); C12N 2750/14121 (2013.01); C12N 2750/14132 (2013.01); C12N 2750/14143 (2013.01); C12N 2830/008 (2013.01);
Abstract

The present invention relates to Adeno-associated virus 9 methods and materials useful for systemically delivering polynucleotides across the blood brain barrier. Accordingly, the present invention also relates to methods and materials useful for systemically delivering polynucleotides to the central and peripheral nervous systems. The present invention also relates to Adeno-associated virus type 9 methods and materials useful for intrathecal delivery of polynucleotides. Use of the methods and materials is indicated, for example, for treatment of lower motor neuron diseases such as spinal muscle atrophy and amyotrophic lateral sclerosis as well as Pompe disease and lysosomal storage disorders. Use of the methods and materials is also indicated, for example, for treatment of Rett syndrome.


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