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Fragile X Syndrome Pre-Clinical Research: Comparing Mouse- and Human-Based Models

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Fragile-X Syndrome

Part of the book series: Methods in Molecular Biology ((MIMB,volume 1942))

Abstract

Despite almost 30 years of biomedical research, a treatment or cure for fragile X syndrome (FXS) is not yet available. The reasons behind this are varied, and among them are discrepancies in both research methodologies and research models. For many years, the fmr1 knockout mouse model dominated the field, and was used to draw important conclusions. The establishment of FXS-human cellular models called these conclusions into question, showing conflicting evidence. Discrepancies in FXS research, between mouse and human, might arise from differences inherent to each species, and from the use of different methodologies. This chapter summarizes these discrepancies and evaluates their impact on the current status of clinical trials.

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Author information

Authors and Affiliations

  1. Department of Molecular and Cell Biology, University of California, Berkeley, CA, USA

    Michael Telias

Authors
  1. Michael Telias
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Corresponding author

Correspondence to Michael Telias .

Editor information

Editors and Affiliations

  1. Wolfe PGD Stem Cell Lab, Racine IVF Unit at Lis Maternity Hospital, Tel Aviv Sourasky Medical Center, Tel Aviv University, Tel Aviv, Israel

    Dalit Ben-Yosef

  2. Wolfe PGD Stem Cell Lab, Racine IVF Unit at Lis Maternity Hospital, Tel Aviv Sourasky Medical Center, Tel Aviv University, Tel Aviv, Israel

    Yoav Mayshar

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Telias, M. (2019). Fragile X Syndrome Pre-Clinical Research: Comparing Mouse- and Human-Based Models. In: Ben-Yosef, D., Mayshar, Y. (eds) Fragile-X Syndrome. Methods in Molecular Biology, vol 1942. Humana Press, New York, NY. https://doi.org/10.1007/978-1-4939-9080-1_13

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  • DOI: https://doi.org/10.1007/978-1-4939-9080-1_13

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  • Publisher Name: Humana Press, New York, NY

  • Print ISBN: 978-1-4939-9079-5

  • Online ISBN: 978-1-4939-9080-1

  • eBook Packages: Springer Protocols

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