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Modeling human neurodegenerative diseases in transgenic systems

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Abstract

Transgenic systems are widely used to study the cellular and molecular basis of human neurodegenerative diseases. A wide variety of model organisms have been utilized, including bacteria (Escherichia coli), plants (Arabidopsis thaliana), nematodes (Caenorhabditis elegans), arthropods (Drosophila melanogaster), fish (zebrafish, Danio rerio), rodents (mouse, Mus musculus and rat, Rattus norvegicus) as well as non-human primates (rhesus monkey, Macaca mulatta). These transgenic systems have enormous value for understanding the pathophysiological basis of these disorders and have, in some cases, been instrumental in the development of therapeutic approaches to treat these conditions. In this review, we discuss the most commonly used model organisms and the methodologies available for the preparation of transgenic organisms. Moreover, we provide selected examples of the use of these technologies for the preparation of transgenic animal models of neurodegenerative diseases, including Alzheimer’s disease (AD), frontotemporal lobar degeneration (FTLD), amyotrophic lateral sclerosis (ALS), Huntington’s disease (HD) and Parkinson’s disease (PD) and discuss the application of these technologies to AD as an example of how transgenic modeling has affected the study of human neurodegenerative diseases.

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Acknowledgments

Supported by the Alzheimer’s Association (IIRG-07-57318), Department of Veterans Affairs (1 I01 BX000342-01) and the National Institute on Aging (P50 AG005138).

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Authors and Affiliations

  1. Research and Development Service, 3F-20, James J. Peters Department of Veterans Affairs Medical Center, 130 West Kingsbridge Road, Bronx, NY, 10468, USA

    Miguel A. Gama Sosa & Rita De Gasperi

  2. Neurology Service, James J. Peters Department of Veterans Affairs Medical Center, Bronx, NY, USA

    Gregory A. Elder

  3. Department of Psychiatry, Mount Sinai School of Medicine of New York University, New York, NY, USA

    Miguel A. Gama Sosa, Rita De Gasperi & Gregory A. Elder

  4. Department of Neurology, Mount Sinai School of Medicine of New York University, New York, NY, USA

    Gregory A. Elder

  5. The Friedman Brain Institute, Mount Sinai School of Medicine of New York University, New York, NY, USA

    Miguel A. Gama Sosa, Rita De Gasperi & Gregory A. Elder

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  1. Miguel A. Gama Sosa
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Gama Sosa, M.A., De Gasperi, R. & Elder, G.A. Modeling human neurodegenerative diseases in transgenic systems. Hum Genet 131, 535–563 (2012). https://doi.org/10.1007/s00439-011-1119-1

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