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US 11,077,208 B2Granted
Wilson's disease gene therapy
AssigneeUCL Business PLC
InventorsAmit Nathwani, Deepak Raj
Life of this patent
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Abstract
There is described a new gene therapy approach for treating Wilson's disease in which a nucleic acid molecule is used which comprises a nucleotide sequence encoding for a functional ATP7B protein wherein the nucleotide sequence has at least 85% identity to the sequence of SEQ ID NO: 1. Also described are vectors comprising the nucleotide sequence and methods and uses thereof.
Claims10
A nucleic acid molecule comprising a nucleotide sequence encoding for a functional ATP7B protein wherein the nucleotide sequence has the sequence of SEQ ID NO.1.
Dependencies are inferred from the claim text.