University of Florida Research Foundation Inc

Antonio L Amelio

David Clair Bloom

Disclosed are genetic expression cassettes, and vectors comprising them useful for the delivery of isolated nucleic acid segments including those expressing or encoding one or more selected therapeutic constructs (including, without limitation, therapeutic peptides, polypeptides, ribozymes, or catalytic RNA molecules), to one or more selected cells or tissues of a vertebrate animal. Methods employing the disclosed genetic constructs in the development of gene therapy-based viral vector systems are also disclosed. The expression cassettes and viral vectors disclosed herein provide new tools for methods of treating mammalian, and in particular, human diseases, disorders, and/or dysfunctions. The disclosed compositions and methods find particular utility in a variety of investigative, diagnostic, and therapeutic regimens, including, for example, in the treatment or amelioration of symptoms of a variety of mammalian, and particularly, human conditions.

Methods for sustained and regulatable gene expression using viral based expression vectors

Disclosed are genetic expression cassettes, and vector comprising them useful for the delivery of nucleic acid segments encoding selected therapeutic constructs (including for example, peptides, polypeptides, ribozymes, and catalytic RN molecules), to selected cells and tissues of vertebrate animals. The disclosed genetic constructs are useful in the development of gene therapy vectors, including for example, viral vectors such as HSV, retroviral, lentiviral, AV, and rAAV vectors. The expression cassettes disclosed herein provide new tools in the field of gene therapy, and for the treatment of mammalian, and in particular, human diseases, disorders, and dysfunctions. The disclosed compositions may be utilized in a variety of investigative, diagnostic and therapeutic regimens, including the prevention and treatment of a variety of human diseases.

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Pittsboro, NC, United States of America

Antonio L Amelio