Oxr1 gene therapy
AssigneeUniversity of Massachusetts
InventorsMichael R. Volkert, Hemant Khanna
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Abstract
Aspects of the disclosure relate to isolated nucleic acids, rAAVs, and compositions configured to express an oxidative stress resistance protein (e.g., OXR1, NCOA7-AS, NCOA7-FL). In some embodiments, the compositions of the disclosure are useful for treatment of diseases or conditions associated with oxidative stress, for example neuronal degeneration.
Claims44
An isolated nucleic acid comprising a transgene comprising a sequence as set forth in any one of SEQ ID NOs:
- 8-14 or 32-40 flanked by two adeno-associated virus (AAV) inverted terminal repeats (ITRs).
An isolated nucleic acid comprising a transgene encoding a protein having an amino acid sequence as set forth in any one of SEQ ID NOs:
- 15-22.
Dependencies are inferred from the claim text.