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Journal Vol 50 (2)

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Large Animal Models of Neurological Disorders for Gene Therapy

Christine Gagliardi and Bruce A. Bunnell

Christine Gagliardi, BS, is a student in the Graduate Program in Biomedical Sciences at Tulane University in New Orleans. Bruce A. Bunnell, PhD, is a professor of pharmacology at the Tulane National Primate Research Center in Covington, Louisiana.

Address correspondence and reprint requests to Dr. Bruce A. Bunnell, Tulane National Primate Research Center, 18703 Three Rivers Road, Covington, LA 70433-8915 or email bbunnell@tulane.edu.

Abstract

The development of therapeutic interventions for genetic disorders and diseases that affect the central nervous system (CNS) has proven challenging. There has been significant progress in the development of gene therapy strategies in murine models of human disease, but gene therapy outcomes in these models do not always translate to the human setting. Therefore, large animal models are crucial to the development of diagnostics, treatments, and eventual cures for debilitating neurological disorders. This review focuses on the description of large animal models of neurological diseases such as lysosomal storage diseases, Parkinson’s disease, Huntington’s disease, and neuroAIDS. The review also describes the contributions of these models to progress in gene therapy research.

Key Words: gene therapy; Huntington’s disease; large animal model; lysosomal storage disease; neuroAIDS; neurologic disease; Parkinson’s disease