Large animal models of neurological disorders for gene therapy

Christine Gagliardi, Bruce A. Bunnell

Research output: Contribution to journalReview articlepeer-review

4 Scopus citations


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.

Original languageEnglish
Pages (from-to)128-143
Number of pages16
JournalILAR Journal
Issue number2
StatePublished - 2009


  • Gene therapy
  • Huntington's disease
  • Large animal model
  • Lysosomal storage disease
  • NeuroAIDS
  • Neurologic disease
  • Parkinson's disease


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