References
Henderson, C. et al. Reversal of disease-related pathologies in the fragile X mouse model by selective activation of GABAB receptors with arbaclofen. Sci. Transl. Med. 4, 152ra128 (2012).
Berry-Kravis, E.M. et al. Effects of STX209 (arbaclofen) on neurobehavioral function in children and adults with fragile X syndrome: a randomized, controlled, phase 2 trial. Sci. Transl. Med. 4, 152ra127 (2012).
Krueger, D.D. & Bear, M.F. Toward fulfilling the promise of molecular medicine in fragile X syndrome. Annu. Rev. Med. 62, 411–429 (2011).
Jacquemont, S. et al. Epigenetic modification of the FMR1 gene in fragile X syndrome is associated with differential response to the mGluR5 antagonist AFQ056. Sci. Transl. Med. 3, 64ra61 (2011).
Soden, M.E. & Chen, L. Fragile X protein FMRP is required for homeostatic plasticity and regulation of synaptic strength by retinoic acid. J. Neurosci. 30, 16910–16921 (2010).
Hall, S.S., Hammond, J.L., Hirt, M. & Reiss, A.L. A. 'learning platform' approach to outcome measurement in fragile X syndrome: a preliminary psychometric study. J. Intellect. Disabil. Res. 56, 947–960 (2012).
Guy, J., Gan, J., Selfridge, J., Cobb, S. & Bird, A. Reversal of neurological defects in a mouse model of Rett syndrome. Science 23, 1143–1147 (2007).
Bear, M.F., Huber, K.M. & Warren, S.T. The mGluR theory of fragile X mental retardation. Trends in Neurosci. 27, 370–377 (2004).
Katz, D.M., et al. Preclinical research in Rett syndrome: setting the foundation for translational success. Dis. Model Mech. 5, 733–745 (2012).
Ecker, C., Spooren, W. & Murphy, D.G. Translational approaches to the biology of Autism: false dawn or a new era? Mol. Psychiatry published online, 10.1038/mp.2012.102 (17 July 2012).
Mendez, M.A. et al. The brain GABA-benzodiazepine receptor alpha-5 subtype in autism spectrum disorder: a pilot [(11)C]Ro15–4513 positron emission tomography study. Neuropharmacology published online, 10.1016/j.neuropharm.2012.04.008 (21 April 2012).
Ecker, C. et al. Describing the brain in autism in five dimensions–magnetic resonance imaging-assisted diagnosis of autism spectrum disorder using a multiparameter classification approach. J. Neurosci. 30, 10612–10623 (2010).
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Trialing targeted therapies for autism. Nat Med 18, 1746–1747 (2012). https://doi.org/10.1038/nm.3027
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DOI: https://doi.org/10.1038/nm.3027
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