Transgene cassettes designed to express a human MECP2 gene
AssigneeBoard of Regents, The University of Texas System
InventorsSteven J. Gray, Sarah Sinnett
Life of this patent
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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.
Claims20
A method for treating Rett Syndrome in a subject in need thereof, comprising administering to the subject an rAAV viral vector, comprising (a) an AAV capsid protein and (b) an rAAV vector comprising, in 5′ to 3′ direction:
- (i) a first AAV ITR sequence comprising the nucleic acid sequence set forth in SEQ ID NO:
- 19;
- (ii) a promoter sequence comprising the nucleic acid sequence set forth in SEQ ID NO:
- 22;
- (iii) a transgene comprising the nucleic acid sequence set forth in SEQ ID NO:
- 3;
- (iv) a regulatory sequence comprising the nucleic acid sequence set forth in SEO ID NO:
- 13;
- and
- (v) a second AAV ITR sequence comprising the nucleic acid sequence set forth in SEQ ID NO:
- 21.
An rAAV vector, comprising, in 5′ to 3′ direction
- (i) a first AAV ITR sequence comprising the nucleic acid sequence set forth in SEQ ID NO:
- 19;
- (ii) a promoter sequence comprising the nucleic acid sequence set forth in SEQ ID NO:
- 22;
- (iii) a transgene encoding a MECP2 polypeptide, the transgene comprising the nucleic acid sequence set forth in SEQ ID NO:
- 3;
- (iv) a regulatory sequence comprising the nucleic acid sequence set forth in SEO ID NO:
- 13;
- and
- (v) a second AAV ITR sequence comprising the nucleic acid sequence set forth in SEQ ID NO:
- 21.
Dependencies are inferred from the claim text.