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. 26, 2025

Filed:

Apr. 10, 2020
Applicants:

University of Massachusetts, Westborough, MA (US);

The Clinic for Special Children, Inc., Strasburg, PA (US);

Inventors:

Guangping Gao, Worcester, MA (US);

Dan Wang, Worcester, MA (US);

Jiaming Wang, Worcester, MA (US);

Kevin A. Strauss, Strasburg, PA (US);

Assignees:

University of Massachusetts, Westborough, MA (US);

The Clinic for Special Children, Inc., Strasburg, PA (US);

Attorney:
Primary Examiner:
Assistant Examiner:
Int. Cl.
CPC ...
C12N 9/02 (2006.01); A61K 48/00 (2006.01); A61P 3/00 (2006.01); C12N 15/86 (2006.01);
U.S. Cl.
CPC ...
C12N 9/0008 (2013.01); A61K 48/0058 (2013.01); A61P 3/00 (2018.01); C12N 15/86 (2013.01); C12Y 102/04004 (2013.01); C12N 2750/14143 (2013.01); C12N 2800/22 (2013.01);
Abstract

In some aspects the disclosure provides compositions and methods for promoting expression of functional BCKDHA protein, which is the E1-alpha subunit of the branched-chain alpha-keto acid (BCAA) dehydrogenase complex, in a subject. In some aspects the disclosure provides compositions and methods for promoting expression of functional BCKDHB protein, which is the E1-beta subunit of the branched-chain alpha-keto acid (BCAA) dehydrogenase complex, in a subject. In some aspects the disclosure provides compositions and methods for promoting expression of functional BCKDHA and BCKDHB proteins, in a subject. In some embodiments, the disclosure provides methods of treating a subject having Maple Syrup Urine Disease (MSUD).


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