Article
Therapeutic Strategies in Fragile X Syndrome: From Bench to Bedside and Back.
Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics - 1 Jul 2015
Gross Christina, Hoffmann Anne, Bassell Gary J, Berry-Kravis Elizabeth M
Abstract excerpt
Fragile X syndrome (FXS), an inherited intellectual disability often associated with autism, is caused by the loss of expression of the fragile X mental retardation protein. Tremendous progress in basic, preclinical, and translational clinical research has elucidated a variety of molecular-, cellular-, and system-level defects in FXS. This has led to the development of several promising therapeutic strategies,...
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