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AssigneesUniversity of Massachusetts, The Clinic for Special Children, Inc.
InventorsGuangping Gao, Dan Wang, Jiaming Wang, Kevin A. Strauss
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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).
1-89. (canceled)
A method for promoting expression of functional BCKDHA protein and BCKDHB protein in a subject, the method comprising administering to the subject an rAAV comprising a capsid containing a nucleic acid engineered to express BCKDHA and BCKDHB in the liver and/or skeletal muscle of the subject, wherein the subject comprises at least one BCKDHA allele or at least one BCKDHB allele having a mutation associated with Maple Syrup Urine Disease (MSUD), wherein the nucleic acid comprises the sequences set forth in SEQ ID NO:
A method for promoting expression of functional BCKDHA protein and BCKDHB protein in a subject, the method comprising administering to the subject an rAAV comprising an AAV9 capsid containing a nucleic acid engineered to express BCKDHA and BCKDHB in the subject, wherein the subject comprises at least one BCKDHA or at least one BCKDHB allele having a mutation associated with Maple Syrup Urine Disease (MSUD), and wherein the nucleic acid comprises the sequences set forth in SEQ ID NO:
A method of treating Maple Syrup Urine Disease in a subject in need thereof, the method comprising administering to the subject an effective amount of an rAAV comprising an AAV9 capsid containing a nucleic acid engineered to express BCKDHA and BCKDHB in the subject, wherein the nucleic acid comprises the sequences set forth in SEQ ID NO:
A method of treating Maple Syrup Urine Disease in a subject in need thereof, the method comprising administering to the subject an effective amount of an rAAV comprising an AAV9 capsid containing a nucleic acid comprising the sequence of SEQ ID NO:
Dependencies are inferred from the claim text.