TY - JOUR
T1 - Translating molecular advances in fragile x syndrome into therapy
T2 - A review
AU - Hagerman, Randi J
AU - Des-Portes, Vincent
AU - Gasparini, Fabrizio
AU - Jacquemont, Sébastien
AU - Gomez-Mancilla, Baltazar
PY - 2014
Y1 - 2014
N2 - 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 utili7ed 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.
AB - 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 utili7ed 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.
UR - http://www.scopus.com/inward/record.url?scp=84899730390&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=84899730390&partnerID=8YFLogxK
U2 - 10.4088/JCP.73r08774
DO - 10.4088/JCP.73r08774
M3 - Article
C2 - 24813413
AN - SCOPUS:84899730390
VL - 75
JO - Journal of Clinical Psychiatry
JF - Journal of Clinical Psychiatry
SN - 0160-6689
IS - 4
ER -