Compositions useful for treating spinal and bulbar muscular atrophy (sbma)
AssigneeThe Trustees of the University of Pennsylvania
InventorsJames M. Wilson, Christian Hinderer, Eileen Workman
Life of this patent
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Abstract
Compositions useful for treatment of Spinal and Bulbar Muscular Atrophy (SBMA) comprising administration of a recombinant adeno-associated virus (rAAV) vector having an AAV capsid and a vector genome comprising a sequence encoding at least one hairpin forming miRNA that comprises a targeting sequence which binds a target site on the mRNA of human androgen receptor, wherein the miRNA inhibits expression of human androgen receptor, is provided. Also provided are compositions containing a rAAV vector and methods of treating SBMA in patient comprising administration of a rAAV vector.
Claims24
An adeno-associated virus (AAV) comprising an AAVhu68 capsid having packaged therein a vector genome, the vector genome comprising an expression cassette comprising a nucleic acid sequence encoding at least one hairpin forming miRNA that comprises a targeting sequence that binds a miRNA target site on the mRNA of human androgen receptor, operably linked to regulatory sequences which direct expression of the nucleic acid sequence in the subject, wherein the miRNA inhibits expression of human androgen receptor, and wherein the expression cassette is flanked by a 5′ AAV ITR and 3′ AAV ITR.
9-11. (canceled)
(canceled)
19-24. (canceled)
An expression cassette comprising a nucleic acid sequence encoding at least one hairpin forming miRNA that comprises a targeting sequence that binds a miRNA target site on the mRNA of human androgen receptor, operably linked to regulatory sequences which direct expression of the nucleic acid sequence in the subject, wherein the miRNA inhibits expression of human androgen receptor, and wherein the expression cassette is flanked by a 5′ AAV ITR and 3′ AAV ITR.
31-33. (canceled)
Dependencies are inferred from the claim text.