Abstract: The subject invention concerns a recombinant adeno-associated virus vector characterized as being capable of delivering and expressing at least one mammalian gene into a genome of a mammalian host cell such that the expression of the gene is regulated in a tissue specific manner by cis-acting regulatory and promoter elements of the gene. A method of using this recombinant adeno-associated virus vector for therapeutic purposes is also provided.
Type:
Grant
Filed:
September 30, 1996
Date of Patent:
December 30, 2003
Assignees:
National Institutes of Health, University of Pittsburgh
Inventors:
Richard Jude Samulski, Christopher E. Walsh, Arthur W. Nienhuis, Johnson M. Liu, Jeffrey L. Miller