Is a
Patent attributes
Patent Jurisdiction
Patent Number
Date of Patent
November 27, 2018
Patent Application Number
14769596
Date Filed
March 13, 2014
Patent Citations Received
Patent Primary Examiner
Patent abstract
A vector having an expression cassette having a hIDUA gene having a sequence of SEQ ID NO: 1 or a sequence at least about 95% identical thereto which encodes a functional human alpha-L-iduronidase is provided. The vector may be production vector or a rAAV8. Also provided are compositions containing these vectors and methods of treating MPSI and the symptoms associated with Hurler, Hurle-Scheie and Scheie syndromes.
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