Of Men and Mice: Modeling the Fragile X Syndrome
- The Fragile X Syndrome (FXS) is one of the most common forms of inherited intellectual disability in all human societies. Caused by the transcriptional silencing of a single gene, the fragile x mental retardation gene FMR1, FXS is characterized by a variety of symptoms, which range from mental disabilities to autism and epilepsy. More than 20 years ago, a first animal model was described, the Fmr1 knock-out mouse. Several other models have been developed since then, including conditional knock-out mice, knock-out rats, a zebrafish and a drosophila model. Using these model systems, various targets for potential pharmaceutical treatments have been identified and many treatments have been shown to be efficient in preclinical studies. However, all attempts to turn these findings into a therapy for patients have failed thus far. In this review, I will discuss underlying difficulties and address potential alternatives for our future research.
Document Type: | Article |
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Language: | English |
Author: | Regina Dahlhaus |
Parent Title (English): | Frontiers in Molecular Neuroscience |
Volume: | 11 |
Article Number: | 41 |
Number of pages: | 38 |
ISSN: | 1662-5099 |
URN: | urn:nbn:de:hbz:1044-opus-36374 |
DOI: | https://doi.org/10.3389/fnmol.2018.00041 |
PMID: | https://pubmed.ncbi.nlm.nih.gov/29599705 |
Publisher: | Frontiers Media |
Place of publication: | Lausanne |
Publishing Institution: | Hochschule Bonn-Rhein-Sieg |
Date of first publication: | 2018/03/15 |
Copyright: | © 2018 Dahlhaus. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). |
Keyword: | E/I balance; FMR1; Fragile X Syndrome; autism spectrum disorders; behavior and cognition; microsatellite instability; mouse model; primates |
Departments, institutes and facilities: | Fachbereich Angewandte Naturwissenschaften |
Dewey Decimal Classification (DDC): | 5 Naturwissenschaften und Mathematik / 57 Biowissenschaften; Biologie / 570 Biowissenschaften; Biologie |
Entry in this database: | 2018/05/04 |
Licence (German): | Creative Commons - CC BY - Namensnennung 4.0 International |