Invention Grant
- Patent Title: Compositions and methods for treating MPSI
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Application No.: US16166346Application Date: 2018-10-22
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Publication No.: US10792343B2Publication Date: 2020-10-06
- Inventor: James M. Wilson , Brittney L. Gurda
- Applicant: The Trustees of the University of Pennsylvania
- Applicant Address: US PA Philadelphia
- Assignee: The Trustees of the University of Pennsylvania
- Current Assignee: The Trustees of the University of Pennsylvania
- Current Assignee Address: US PA Philadelphia
- Agency: Howson & Howson LLP
- Agent Cathy A. Kodroff
- Main IPC: A61K38/47
- IPC: A61K38/47 ; C12N9/24 ; A61K48/00 ; A61K9/00 ; A61K35/76 ; C12N7/00

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.
Public/Granted literature
- US20190111116A1 COMPOSITIONS AND METHODS FOR TREATING MPSI Public/Granted day:2019-04-18
Information query
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