Patent ID: 11958887
Assignee: THE GENERAL HOSPITAL CORPORATION
Field: Pharmaceuticals (Chemistry)
Classification: CPC A  C | IPC A  C

Claim 12:
13. A recombinant adeno-associated virus (rAAV), said rAAV comprising an AAV capsid and an AAV genome packaged therein, said AAV genome comprising a nucleic acid molecule capable of expressing a protein comprising a hamartin binding region and a GAP region, wherein said protein comprises: (i) a hamartin binding region having at least 94% sequence identity to SEQ ID NO: 2, (ii) a GTPase-activating protein (GAP) region having at least 93% sequence identity to SEQ ID NO: 3, and (iii) a spacer sequence linking the hamartin binding region to the GAP region, wherein the spacer sequence comprises SGGG (SEQ ID NO: 13), and wherein said protein lacks amino acids 451-1514 of human tuberin (SEQ ID NO: 10).