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.
Claims17
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;
- (ii) a promoter sequence;
- (iii) a transgene comprising a nucleic acid sequence encoding a MeCP2 polypeptide;
- (iv) a regulatory sequence comprising a sequence with at least 80% identity to the nucleic acid sequence set forth in SEO ID NO:
- 13;
- and
- (v) a second AAV ITR sequence.
Dependencies are inferred from the claim text.