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Large animal models of neurological disorders for gene therapy
Christine Gagliardi1, Bruce A Bunnell
1Tulane National Primate Research Center, Covington, LA 70433-8915, USA.
Abstract:
he 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, Parkinsons disease, Huntingtons disease, and neuroAIDS. The review also describes the contributions of these models to progress in gene therapy research.
Insights
Large animal models are vital for advancing gene therapy for central nervous system (CNS) diseases. These models improve the translation of therapeutic strategies from research to human clinical applications for neurological disorders.
Area of Science:
- Neuroscience
- Genetics
- Translational Medicine
Background:
- Developing treatments for central nervous system (CNS) genetic disorders is challenging.
- Murine models show promise for gene therapy but often fail to translate to human patients.
- Large animal models are essential for bridging this translational gap.
Purpose of the Study:
- To review the utility of large animal models in neurological disease research.
- To highlight their role in advancing gene therapy for CNS disorders.
- To discuss specific disease models including lysosomal storage diseases, Parkinson's disease, Huntington's disease, and neuroAIDS.
Main Methods:
- Literature review of studies utilizing large animal models for neurological diseases.
- Analysis of gene therapy research progress in these models.
- Description of established large animal models for specific neurodegenerative and genetic disorders.
Main Results:
- Large animal models offer a more translatable platform for studying complex neurological diseases.
- These models have significantly contributed to the development and testing of gene therapy strategies.
- Specific examples illustrate the successful application of these models in Parkinson's, Huntington's, and lysosomal storage diseases.
Conclusions:
- Large animal models are indispensable for the development of effective diagnostics and therapeutics for neurological disorders.
- Continued use and development of these models will accelerate progress in gene therapy for CNS diseases.
- Translational research using large animals is key to finding cures for debilitating neurological conditions.