Transgene cassettes designed to express a human MECP2 gene
AssigneeBoard of Regents, The University of Texas System
InventorsSteven J. Gray, Sarah Sinnett
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Abstract
The present disclosure provides methods and compositions for the treatment of diseases and genetic disorders linked to MeCP2 loss and/or misfunction, including RETT syndrome. The methods and compositions of the present disclosure comprise rAAV vectors and rAAV viral vectors comprising transgene nucleic acid molecules comprising nucleic acid sequences encoding for a MeCP2 polypeptide.
Claims31
An rAAV vector, comprising, in a 5′ to 3′ direction:
- a) a first AAV ITR sequence;
- b) a promoter sequence;
- c) a transgene nucleic acid molecule, wherein the transgene nucleic acid molecule comprises a nucleic acid sequence encoding for an MeCP2-derived polypeptide,
- wherein the MeCP2-derived polypeptide is a miniMeCP2 polypeptide;
- d) a regulatory sequence comprising a sequence with at least 80% identity to the nucleic acid sequence set forth in SEQ ID NO:13;
- and
- e) a second AAV ITR sequence.
An rAAV vector, comprising, in a 5′ to 3′ direction:
- a) a first AAV ITR sequence;
- b) a promoter sequence comprising an MeP426 promoter sequence;
- c) a transgene nucleic acid molecule, wherein the transgene nucleic acid molecule comprises a nucleic acid sequence encoding for an MeCP2-derived polypeptide;
- d) a regulatory sequence comprising the nucleic acid sequence set forth in SEQ ID NO:
- 17;
- and
- e) a second AAV ITR sequence.
An rAAV vector, comprising, in a 5′ to 3′ direction:
- a) a first AAV ITR sequence;
- b) a promoter sequence comprising an MeP426 promoter sequence;
- c) a transgene nucleic acid molecule, wherein the transgene nucleic acid molecule comprises a nucleic acid sequence encoding for an MeCP2-derived polypeptide;
- d) a regulatory sequence comprising the nucleic acid sequence set forth in SEQ ID NO:
- 14;
- and
- e) a second AAV ITR sequence.
An rAAV vector comprising at least 80% identity to the nucleic acid sequence set forth in SEQ ID NO:
- 29.
Dependencies are inferred from the claim text.