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filingDate 2016-12-19-04:00^^<http://www.w3.org/2001/XMLSchema#date>
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publicationDate 2017-06-22-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber WO-2017103624-A1
titleOfInvention Wilson's disease gene therapy
abstract There is described a 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.
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