Compositions useful for treating GM1 gangliosidosis
AssigneeThe Trustees of the University of Pennylvania
InventorsJames M. Wilson, Christian Hinderer, Nathan Katz
Life of this patent
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Abstract
A recombinant adeno-associated virus (rAAV) comprising an AAVhu68 capsid and a vector genome comprising a lysosomal beta-galactosidase gene (for example, galactosidase beta 1 gene, GBL1) is provided (i.e., rAAVhu68.GBL1). Also provided a composition containing an effective amount of rAAVhu68.GBL1 to ameliorate symptoms of GM1 gangliosidosis, including, e.g., increased average life span, decreased need for feeding tube, reduction in seizure incidence and frequency, reduction in progression towards neurocognitive decline and/or improvement in neurocognitive development.
Claims17
An adeno-associated virus (AAV) having an AAVhu68 capsid and a vector genome comprising a β-galactosidase 1 (GLB1) gene encoding human β-galactosidase operably linked to regulatory sequences, wherein the GLB1 gene comprises SEQ ID NO:
- 8 or a sequence at least 95% identical to SEQ ID NO:
- 8 that encodes human β-galactosidase comprising amino acids 1 to 677 of SEQ ID NO:
- 4.
An adeno-associated virus (AAV) having an AAVhu68 capsid and a vector genome comprising a β-galactosidase 1 (GLB1) gene encoding human β-galactosidase operably linked to regulatory sequences which comprise a human ubiquitin C (UbC) promoter, wherein the GLB1 gene comprises SEQ ID NO:
- 8 or a sequence at least 99% identical to SEQ ID NO:
- 8 that encodes human β-galactosidase comprising amino acids 1 to 677 of SEQ ID NO:
- 4.
Dependencies are inferred from the claim text.