COMPOSITIONS ET METHODES DE TRAITEMENT DE LA MPS1

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专利权人:

THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA

发明人:

WILSON, JAMES M.,GURDA, BRITTNEY L.

申请号：

CA2901328

公开号：

CA2901328A1

申请日:

2014.03.13

申请国别(地区):

CA

年份:

2014

代理人:

摘要：

A vector have an expression cassette containing have a hIDUA gene has 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 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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