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filingDate 2020-09-10-04:00^^<http://www.w3.org/2001/XMLSchema#date>
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publicationDate 2020-12-24-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber US-2020397872-A1
titleOfInvention Compositions and methods for treating mpsi
abstract A vector have an expression cassette containing have a hIDUA gene has a sequence of SEQ ID NO: 1 (FIG. 1 ) or a sequence at least about 95% identical thereto which encodes a functional human alpha-L-iduronidase is provided. The vector may be a production vector or a rAAV8. Also provided are compositions containing these vectors and methods of treating MPSI and the symptoms associated with Hurler, Hurler-Scheie and Scheie syndromes.
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