Frataxin expression constructs
AssigneePrecigen, Inc.
InventorsStephen Schauer, Darby Thomas, Gregory Robinson, Mark Pykett, Richard Thorn, Kirsten Gruis
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Abstract
The invention provides polynucleotides, vectors and viruses expressing frataxin and methods of treating Friedreich's Ataxia.
Claims23
A polynucleotide comprising a nucleic acid molecule encoding a human frataxin polypeptide having an amino acid sequence of SEQ ID NO:1, wherein the nucleic acid molecule is operably linked to a 5′UTR and a 3′UTR, wherein:
- the 5′UTR is RPL6-5′Splice and the 3′UTR is a synthetic 3′ regulatory element having a nucleic acid sequence of SEQ ID NO:7;
- or
- the 5′UTR is 5U2 and the 3′UTR is a synthetic 3′ regulatory element having a nucleic acid sequence of SEQ ID NO:7,
- wherein the encoded human frataxin polypeptide is a functional frataxin protein.
A recombinant virion which comprises a viral vector, wherein said vector comprises a nucleic acid molecule encoding human frataxin operably linked to a 5′UTR, a 3′UTR, and a control element that directs transcription and/or translation, wherein:
- the 5′UTR is RPL6-5′Splice and the 3′UTR is a synthetic 3′ regulatory element having a nucleic acid sequence of SEQ ID NO:7;
- or
- the 5′UTR is 5U2 and the 3′UTR is a synthetic 3′ regulatory element having a nucleic acid sequence of SEQ ID NO:7.
A composition comprising:
- (a) a viral vector, wherein said vector comprises a nucleic acid molecule encoding human frataxin operably linked to a 5′UTR, a 3′UTR, and a control element that directs transcription and/or translation, wherein:
- the 5′UTR is RPL6-5′Splice and the 3′UTR is a synthetic 3′ regulatory element having a nucleic acid sequence of SEQ ID NO:7;
- or
- the 5′UTR is 5U2 and the 3′UTR is a synthetic 3′ regulatory element having a nucleic acid sequence of SEQ ID NO:7;
- and
- (b) a pharmaceutically acceptable excipient.
Dependencies are inferred from the claim text.