AAV-mediated gene therapy for maple syrup urine disease (MSUD)
US12398378B2 · kind B2 · utility
Assignees
Inventors
Key dates
| Filing date | Apr 10, 2020 |
| Grant date | Aug 26, 2025 |
| Priority date | — |
| Expiry date | Nov 28, 2042 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N9/0008
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
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).
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.