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:
Feb. 14, 2023

Filed:

Feb. 27, 2018
Applicant:

The Trustees of the University of Pennsylvania, Philadelphia, PA (US);

Inventors:

James M. Wilson, Philadelphia, PA (US);

Christian Hinderer, Philadelphia, PA (US);

Nathan Katz, Stamford, CT (US);

Qiang Wang, Philadelphia, PA (US);

Assignee:
Attorneys:
Primary Examiner:
Int. Cl.
CPC ...
A61K 48/00 (2006.01); C12N 15/86 (2006.01); C07K 14/47 (2006.01); C12N 15/864 (2006.01); A61K 45/06 (2006.01);
U.S. Cl.
CPC ...
C12N 15/8645 (2013.01); C07K 14/47 (2013.01); C12N 15/864 (2013.01); A61K 45/06 (2013.01); C12N 2750/14143 (2013.01); C12N 2800/22 (2013.01);
Abstract

A rAAV vector is described herein which has an AAVhu68 capsid and at least one expression cassette in the capsid. The at least one expression cassette comprises nucleic acid sequences encoding a functional SMN protein and expression control sequences that direct expression of the SMN sequences in a host cell. Also provided are compositions containing this rAAVhu68.SMN vector and methods of using same for spinal muscular atrophy in a patient.


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