The University of North Carolina at Chapel Hill

University of Florida Research Foundation, Incorporated

University of Pennsylvania

Brittney Gurda

Mavis Agbandje-McKenna

Aravind Asokan

Long Ping Victor Tse

James M Wilson

The present invention provides AAV capsid proteins comprising a modification in the amino acid sequence and virus vectors comprising the modified AAV capsid protein. The invention also provides methods of administering the virus vectors and virus capsids of the invention to a cell or to a subject in vivo.

Methods and compositions for antibody-evading virus vectors

A vector have an expression cassette containing have a hIDUA gene has a sequence of SEQ ID NO: 1 (FIG.) 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.

Compositions and methods for treating MPSI

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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Collegeville, PA, United States of America

Brittney Gurda