Article: article from journal or magazin.
Review (review): journal as complete as possible of one specific subject, written based on exhaustive analyses from published work.
Translating molecular advances in fragile X syndrome into therapy: a review.
Journal of Clinical Psychiatry
Publication types: Journal Article ; Research Support, Non-U.S. Gov't ; Review Publication Status: ppublish Document Type: Review PDF : Review Article
Fragile X syndrome is an inherited disease with cognitive, behavioral, and neurologic manifestations, resulting from a single genetic mutation. A variety of treatments that target individual symptoms of fragile X syndrome are currently utilized with limited efficacy. Research in animal models has resulted in the development of potential novel pharmacologic treatments that target the underlying molecular defect in fragile X syndrome, rather than the resultant symptoms. This review describes recent advances in our understanding of the molecular basis of fragile X syndrome and summarizes the ongoing clinical research programs.
Animals, Biomedical Research, Fragile X Mental Retardation Protein/genetics, Fragile X Syndrome/drug therapy, Fragile X Syndrome/etiology, Humans, Mice, Mice, Knockout/genetics, Receptor, Metabotropic Glutamate 5/drug effects, Receptor, Metabotropic Glutamate 5/genetics, Receptors, GABA/drug effects, Receptors, GABA/metabolism
Web of science
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