University of South Florida

Siddharth G Kamath

Chad Dickey

Basil Cherpelis

Lori Lebson

Dave Morgan

The subject invention concerns materials and methods for conversion of non-neuronal cells into neurons. The subject invention also concerns methods for screening drugs and other compounds for activity in treating Alzheimer's disease using neuronal cells produced using the subject invention. The subject invention also concerns neuronal cells that have been produced using the methods of the invention. The subject invention also concerns methods for evaluating therapeutic treatment for efficacy in a person or animal having Alzheimer's disease or other neurodegenerative diseases. The subject invention also concerns methods of treating Alzheimer's disease or other neurodegenerative diseases or conditions in a person or animal.

Conversion of non-neuronal cells into neurons

Disclosed is a method for the treatment of a neurodegenerative disorder by isolating a plurality of monocytic cells and introducing a vector containing a gene that expresses a protease capable of degrading amyloid peptide into the isolated monocytic cells. The modified cells are then administered to the patient. Preferably the introduced gene is selected from the group consisting of neprilysin, insulin degrading enzyme and endothelin converting enzyme. The protease capable of degrading amyloid peptide is secreted from the monocytic cells into the extracellular space thereof. According to one variant, the gene that expresses a protease capable of degrading amyloid peptide is the NEP gene with a deletion in the membrane binding domain and/or an appended signal peptide to drive secretion of the modified gene product. This invention contemplates the use of heterologous and autologos (the monocytic cells are obtained from the patient) transplants.

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Tampa, FL, United States of America

Siddharth G Kamath