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US 10,703,797 B2Granted
Gene therapy vectors for treatment of Danon disease
AssigneeRocket Pharmaceuticals, Ltd.
InventorsAnnahita Keravala, Simon Moore, David Ricks
Life of this patent
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Abstract
The disclosure relates to optimized polynucleotide sequences for LAMP-2B, expression cassettes, vectors, and methods of use thereof in treating disease, e.g. Danon disease.
Claims15
An adeno-associated virus (AAV) vector, comprising an expression cassette comprising a polynucleotide sequence that encodes a LAMP-2B protein, operatively linked to a promoter, wherein the polynucleotide sequence comprises any one of SEQ ID NOs:
- 3-5.
A polynucleotide sequence comprising any one of SEQ ID NOs:
- 3-5.
Dependencies are inferred from the claim text.